부산대학교 도서관

Bettini, S.; Schiff, S.; Callegari, C.; Gusella, B.; Pontesilli, G.M.; D’Angelo, M.; Baldan, V.; Zattarin, A.; Romanelli, G.; Angeli, P.; Spinella, P.; Vettor, R.; Busetto, L.; Carraro, E.; Girardi, P.

In: Nutrients. (Nutrients, December 2023, 15(23))

Pontesilli, Martina ; Painter, Rebecca C.; Grooten, Iris J.; van der Post, Joris A.; Mol, Ben Willem; Vrijkotte, Tanja G.M.; Repping, Sjoerd; Roseboom, Tessa J.

In Reproductive BioMedicine Online March 2015 30(3):258-267

Jaramillo-Gutierrez, Giovanna; Benschop, Kimberley S.M.; Claas, Eric C.J.; de Jong, Arjan S.; van Loon, Anton M.; Pas, Suzan D.; Pontesilli, Oscar; Rossen, John W.; Swanink, Caroline M.A.; Thijsen, Steven; van der Zanden, Adri G.M.; van der Avoort, Harrie G.A.M.; Koopmans, Marion P.G.; Meijer, Adam

In Journal of Virological Methods June 2013 190(1-2):53-62

Garelli, S.; Salituro, N.; Pontesilli, G.M.; Ricciardiello, L.; Vicennati, V.; Pagotto, U.

In: Encyclopedia of Endocrine Diseases. (Encyclopedia of Endocrine Diseases, 1 January 2018, :464-472)

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