학술논문

발행년
-
(예 : 2010-2015)
'학술논문' 에서 검색결과 148건 | 목록 1~10
Academic Journal
Consiglieri G; Pediatric Immunohematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.; Department of Systems Medicine, University of Rome Tor Vergata, 00133 Rome, Italy.; Tucci F; Pediatric Immunohematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.; De Pellegrin M; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.; Guerrini B; 'Vita-Salute' San Raffaele University, 20132 Milan, Italy.; Cattoni A; Paediatrics, IRCCS San Gerardo dei Tintori Foundation, 20900 Monza, Italy.; School of Medicine and Surgery, University of Milano-Bicocca, 20126 Milan, Italy.; Risca G; Bicocca Bioinformatics Biostatistics and Bioimaging B4 Center, School of Medicine and Surgery, University of Milano-Bicocca, 20854 Vedano al Lambro, Italy.; Scarparo S; Pediatric Immunohematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.; Sarzana M; Pediatric Immunohematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.; Pontesilli S; Neuroradiology Unit, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.; Mellone R; Radiology Unit, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.; Gasperini S; Paediatrics, IRCCS San Gerardo dei Tintori Foundation, 20900 Monza, Italy.; Galimberti S; Bicocca Bioinformatics Biostatistics and Bioimaging B4 Center, School of Medicine and Surgery, University of Milano-Bicocca, 20854 Vedano al Lambro, Italy.; Units of Neurology and Neurophysiology, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.; Silvani P; Anesthesia and Critical Care, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.; Filisetti C; Pediatric Immunohematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.; 'Vita-Salute' San Raffaele University, 20132 Milan, Italy.; Darin S; Pediatric Immunohematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.; Forni G; Department of Experimental and Clinical Biomedical Sciences, University of Florence, 50134 Florence, Italy.; Miglietta S; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.; Santi L; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.; Facchini M; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.; Corti A; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.; Fumagalli F; Pediatric Immunohematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.; Cicalese MP; Pediatric Immunohematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.; 'Vita-Salute' San Raffaele University, 20132 Milan, Italy.; Calbi V; Pediatric Immunohematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.; Migliavacca M; Pediatric Immunohematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.; Barzaghi F; Pediatric Immunohematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.; Ferrua F; Pediatric Immunohematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.; Gallo V; Pediatric Immunohematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.; Recupero S; Pediatric Immunohematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.; Canarutto D; Pediatric Immunohematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.; Doglio M; Pediatric Immunohematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.; Tedesco L; Paediatrics, IRCCS San Gerardo dei Tintori Foundation, 20900 Monza, Italy.; Volpi N; Department of Life Sciences, University of Modena and Reggio Emilia, Laboratory of Biochemistry and Glycobiology, 41125 Modena, Italy.; Rovelli A; Paediatrics, IRCCS San Gerardo dei Tintori Foundation, 20900 Monza, Italy.; la Marca G; Department of Experimental and Clinical Biomedical Sciences, University of Florence, 50134 Florence, Italy.; Newborn Screening, Clinical Chemistry and Pharmacology Laboratory, Meyer Children's Hospital IRCCS, 50139 Florence, Italy.; Valsecchi MG; Bicocca Bioinformatics Biostatistics and Bioimaging B4 Center, School of Medicine and Surgery, University of Milano-Bicocca, 20854 Vedano al Lambro, Italy.; Biostatistics and Clinical Epidemiology, Fondazione IRCCS San Gerardo dei Tintori, 20900 Monza, Italy.; Zancan S; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.; Ciceri F; 'Vita-Salute' San Raffaele University, 20132 Milan, Italy.; Department of Haematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.; Naldini L; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.; 'Vita-Salute' San Raffaele University, 20132 Milan, Italy.; Baldoli C; Neuroradiology Unit, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.; Parini R; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.; Gentner B; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.; Ludwig Institute for Cancer Research and Department of Oncology, University of Lausanne (UNIL) and Lausanne University Hospital (CHUV), 1015 Lausanne, Switzerland.; Aiuti A; Pediatric Immunohematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.; 'Vita-Salute' San Raffaele University, 20132 Milan, Italy.; Bernardo ME; Pediatric Immunohematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.; 'Vita-Salute' San Raffaele University, 20132 Milan, Italy.
Publisher: American Association for the Advancement of Science Country of Publication: United States NLM ID: 101505086 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1946-6242 (Electronic) Linking ISSN: 19466234 NLM ISO Abbreviation: Sci Transl Med Subsets: MEDLINE
Academic Journal
Rovelli A; Department of Chemical Engineering, Imperial College London, South Kensington, London SW7 2AZ, U.K.; Brodie J; BP International Ltd, Chertsey Road, Sunbury-on-Thames TW16 7LN, U.K.; Rashid B; BP International Ltd, Chertsey Road, Sunbury-on-Thames TW16 7LN, U.K.; Tay WJ; BP International Ltd, Chertsey Road, Sunbury-on-Thames TW16 7LN, U.K.; Pini R; Department of Chemical Engineering, Imperial College London, South Kensington, London SW7 2AZ, U.K.
Publisher: The Society Country of Publication: United States NLM ID: 100971627 Publication Model: eCollection Cited Medium: Print ISSN: 0887-0624 (Print) Linking ISSN: 08870624 NLM ISO Abbreviation: Energy Fuels Subsets: PubMed not MEDLINE
Academic Journal
Napolitano S; Hematology-Oncology and Bone Marrow Transplantation Unit, Pediatric Department, and Monza and Brianza Foundation for Children and their Mums, San Gerardo Hospital, Lombardia, Monza, Italy.; Ottaviano G; Molecular and Cellular Immunology Unit, University College of London, Great Ormond Street Institute of Child Health, London, United Kingdom.; Bettini L; Tettamanti Laboratory, Monza and Brianza Foundation for Children and their Mums, Monza, Lombardia, Italy.; Russotto V; Department of Emergency and Intensive Care, University Hospital San Gerardo, Monza, Italy.; Bonanomi S; Hematology-Oncology and Bone Marrow Transplantation Unit, Pediatric Department, and Monza and Brianza Foundation for Children and their Mums, San Gerardo Hospital, Lombardia, Monza, Italy.; Rovelli A; Hematology-Oncology and Bone Marrow Transplantation Unit, Pediatric Department, and Monza and Brianza Foundation for Children and their Mums, San Gerardo Hospital, Lombardia, Monza, Italy.; Biondi A; Hematology-Oncology and Bone Marrow Transplantation Unit, Pediatric Department, and Monza and Brianza Foundation for Children and their Mums, San Gerardo Hospital, Lombardia, Monza, Italy.; University of Milan-Bicocca, Monza, Italy.; Rona R; Department of Emergency and Intensive Care, University Hospital San Gerardo, Monza, Italy.; Balduzzi A; Hematology-Oncology and Bone Marrow Transplantation Unit, Pediatric Department, and Monza and Brianza Foundation for Children and their Mums, San Gerardo Hospital, Lombardia, Monza, Italy.; University of Milan-Bicocca, Monza, Italy.
Publisher: Sage Publications Country of Publication: United States NLM ID: 0111356 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2038-2529 (Electronic) Linking ISSN: 03008916 NLM ISO Abbreviation: Tumori Subsets: MEDLINE
Academic Journal
Cattoni A; Department of Pediatrics, Università degli Studi di Milano-Bicocca, Fondazione MBBM, Ospedale San Gerardo, Monza, Italy. Electronic address: alessandro.cattoni@unimib.it.; Molinari S; Department of Pediatrics, Università degli Studi di Milano-Bicocca, Fondazione MBBM, Ospedale San Gerardo, Monza, Italy.; Gaiero A; Department of Pediatrics, Ospedale San Paolo, Savona, Italy.; De Lorenzo P; Tettamanti Research Center, Department of Pediatrics, Università degli Studi di Milano-Bicocca, Monza, Italy; Bicocca Center of Bioinformatics, Biostatistics and Bioimaging (B4), School of Medicine and Surgery, Università degli Studi di Milano-Bicocca, Monza, Italy.; Fichera G; Department of Pediatrics, Ospedale San Paolo, Savona, Italy.; Riva B; Department of Pediatrics, Università degli Studi di Milano-Bicocca, Fondazione MBBM, Ospedale San Gerardo, Monza, Italy.; Di Marco S; Department of Pediatrics, Università degli Studi di Milano-Bicocca, Fondazione MBBM, Ospedale San Gerardo, Monza, Italy.; Tommesani C; Department of Pediatrics, Università degli Studi di Milano-Bicocca, Fondazione MBBM, Ospedale San Gerardo, Monza, Italy.; Mariani E; Department of Pediatrics, Università degli Studi di Milano-Bicocca, Fondazione MBBM, Ospedale San Gerardo, Monza, Italy.; Medici F; Department of Pediatrics, Università degli Studi di Milano-Bicocca, Fondazione MBBM, Ospedale San Gerardo, Monza, Italy.; Pagni F; Department of Pathology, Università degli Studi di Milano-Bicocca, Ospedale San Gerardo, Monza, Italy.; Nicolosi ML; Department of Pediatrics, Università degli Studi di Milano-Bicocca, Fondazione MBBM, Ospedale San Gerardo, Monza, Italy.; Fraschini D; Department of Pediatrics, Università degli Studi di Milano-Bicocca, Fondazione MBBM, Ospedale San Gerardo, Monza, Italy.; Napolitano S; Department of Pediatrics, Università degli Studi di Milano-Bicocca, Fondazione MBBM, Ospedale San Gerardo, Monza, Italy.; Rovelli A; Department of Pediatrics, Università degli Studi di Milano-Bicocca, Fondazione MBBM, Ospedale San Gerardo, Monza, Italy.; Biondi A; Department of Pediatrics, Università degli Studi di Milano-Bicocca, Fondazione MBBM, Ospedale San Gerardo, Monza, Italy.; Valsecchi MG; Bicocca Center of Bioinformatics, Biostatistics and Bioimaging (B4), School of Medicine and Surgery, Università degli Studi di Milano-Bicocca, Monza, Italy.; Balduzzi A; Bicocca Center of Bioinformatics, Biostatistics and Bioimaging (B4), School of Medicine and Surgery, Università degli Studi di Milano-Bicocca, Monza, Italy.
Publisher: Elsevier Inc Country of Publication: United States NLM ID: 101774629 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2666-6367 (Electronic) Linking ISSN: 26666367 NLM ISO Abbreviation: Transplant Cell Ther Subsets: MEDLINE
Academic Journal
Pontesilli S; Department of Neuroradiology, IRCCS San Raffaele Scientific Institute, Milano, Italy.; Baldoli C; Department of Neuroradiology, IRCCS San Raffaele Scientific Institute, Milano, Italy.; Rosa PAD; Department of Neuroradiology, IRCCS San Raffaele Scientific Institute, Milano, Italy.; Cattoni A; Pediatric Department, Fondazione MBBM, San Gerardo Hospital, University of Milano-Bicocca, Monza, Italy.; Bernardo ME; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milano, Italy; Vita-Salute San Raffaele University of Milano, Milano, Italy.; Meregalli P; Pediatric Department, Fondazione MBBM, San Gerardo Hospital, University of Milano-Bicocca, Monza, Italy.; Gasperini S; Pediatric Department, Fondazione MBBM, San Gerardo Hospital, University of Milano-Bicocca, Monza, Italy.; Motta S; Pediatric Department, Fondazione MBBM, San Gerardo Hospital, University of Milano-Bicocca, Monza, Italy.; Fumagalli F; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milano, Italy.; Tucci F; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milano, Italy.; Baciga F; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milano, Italy.; Consiglieri G; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milano, Italy.; Canonico F; Department of Radiology, San Gerardo Hospital, University of Milano-Bicocca, Monza, Italy.; De Lorenzo P; Centro Operativo di Ricerca Statistica, Fondazione Tettamanti, University of Milano-Bicocca, Monza, Italy.; Chiapparini L; Neuroradiology Unit, IRCCS Istituto Neurologico Carlo Besta, Milano, Italy.; Gentner B; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milano, Italy; Vita-Salute San Raffaele University of Milano, Milano, Italy.; Aiuti A; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milano, Italy; Vita-Salute San Raffaele University of Milano, Milano, Italy.; Biondi A; Pediatric Department, Fondazione MBBM, San Gerardo Hospital, University of Milano-Bicocca, Monza, Italy.; Rovelli A; Pediatric Department, Fondazione MBBM, San Gerardo Hospital, University of Milano-Bicocca, Monza, Italy.; Parini R; Pediatric Department, Fondazione MBBM, San Gerardo Hospital, University of Milano-Bicocca, Monza, Italy; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milano, Italy. Electronic address: rossella.parini@unimib.it.
Publisher: Mosby Country of Publication: United States NLM ID: 0375410 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1097-6833 (Electronic) Linking ISSN: 00223476 NLM ISO Abbreviation: J Pediatr Subsets: MEDLINE
Academic Journal
Ottaviano G; Bone Marrow Transplant Unit, Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom. Electronic address: g.ottaviano@ucl.ac.uk.; Achini-Gutzwiller F; Department of Pediatrics, Stem Cell Transplantation Program, Willem-Alexander Children's Hospital, Leiden University Medical Center, Leiden, Netherlands; Division of Pediatric Stem Cell Transplantation and Children's Research Center (CRC), University Children's Hospital of Zurich, Zurich, Switzerland.; Kalwak K; Department and Clinic of Pediatric Hematology, Oncology and Bone Marrow Transplantation, Wroclaw Medical University, Wroclaw, Poland.; Lanino E; Istituto G. Gaslini, Hematopoietic Stem Cell Transplantation Unit- Hematology-Oncology, Genova, Italy.; Faraci M; Istituto G. Gaslini, Hematopoietic Stem Cell Transplantation Unit- Hematology-Oncology, Genova, Italy.; Rao K; Bone Marrow Transplant Unit, Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom.; Chiesa R; Bone Marrow Transplant Unit, Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom.; Amrolia P; Bone Marrow Transplant Unit, Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom.; Bonanomi S; Clinica Pediatrica, Università degli studi di Milano-Bicocca, Ospedale San Gerardo/Fondazione MBBM, Monza, Italy.; Rovelli A; Clinica Pediatrica, Università degli studi di Milano-Bicocca, Ospedale San Gerardo/Fondazione MBBM, Monza, Italy.; Veys P; Bone Marrow Transplant Unit, Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom.; Lankester A; Department of Pediatrics, Stem Cell Transplantation Program, Willem-Alexander Children's Hospital, Leiden University Medical Center, Leiden, Netherlands.; Balduzzi A; Clinica Pediatrica, Università degli studi di Milano-Bicocca, Ospedale San Gerardo/Fondazione MBBM, Monza, Italy.; Lucchini G; Bone Marrow Transplant Unit, Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom.
Publisher: Elsevier Inc Country of Publication: United States NLM ID: 101774629 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2666-6367 (Electronic) Linking ISSN: 26666367 NLM ISO Abbreviation: Transplant Cell Ther Subsets: MEDLINE
Academic Journal
Gentner B; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Tucci F; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Galimberti S; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Fumagalli F; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; De Pellegrin M; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Silvani P; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Camesasca C; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Pontesilli S; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Darin S; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Ciotti F; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Sarzana M; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Consiglieri G; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Filisetti C; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Forni G; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Passerini L; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Tomasoni D; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Cesana D; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Calabria A; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Spinozzi G; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Cicalese MP; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Calbi V; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Migliavacca M; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Barzaghi F; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Ferrua F; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Gallo V; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Miglietta S; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Zonari E; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Cheruku PS; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Forni C; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Facchini M; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Corti A; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Gabaldo M; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Zancan S; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Gasperini S; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Rovelli A; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Boelens JJ; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Jones SA; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Wynn R; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Baldoli C; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Montini E; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Gregori S; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Ciceri F; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Valsecchi MG; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; la Marca G; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Parini R; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Naldini L; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Aiuti A; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.; Bernardo ME; From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
Publisher: Massachusetts Medical Society Country of Publication: United States NLM ID: 0255562 Publication Model: Print Cited Medium: Internet ISSN: 1533-4406 (Electronic) Linking ISSN: 00284793 NLM ISO Abbreviation: N Engl J Med Subsets: MEDLINE
검색 결과 제한하기
제한된 항목
[AR] Rovelli A
발행연도 제한
-
학술DB(Database Provider)
저널명(출판물, Title)
출판사(Publisher)
자료유형(Source Type)
주제어
언어