부산대학교 도서관

Musolino, Patricia L; Rosser, Susan J; Brittan, Mairi; Newby, David E; Berry, Colin; Riley, Paul R; Giacca, Mauro; Hajjar, Roger J; Baker, Andrew H

Cardiovascular Research. Oct2025, Vol. 121 Issue 12, p1843-1855. 13p.

Pandiri S; The University of Missouri Kansas City School of Medicine, Kansas City, Missouri and.; Chaaya C; The Department of Ophthalmology, Massachusetts Eye and Ear.; Meshkin RS; The Department of Ophthalmology, Massachusetts Eye and Ear.; Hoyek S; The Department of Ophthalmology, Massachusetts Eye and Ear.; Rizzo JF 3rd; The Department of Ophthalmology, Massachusetts Eye and Ear.; Tambala D; Department of Neurology, Center for Genomic Medicine, Massachusetts General Hospital and.; Cruz ED; Department of Neurology, Center for Genomic Medicine, Massachusetts General Hospital and.; Lynch A; Department of Neurology, Center for Genomic Medicine, Massachusetts General Hospital and.; Gise R; The Department of Ophthalmology, Massachusetts Eye and Ear.; Musolino PL; Department of Neurology, Center for Genomic Medicine, Massachusetts General Hospital and.; Harvard Medical School, Boston, Massachusetts.; Patel NA; The Department of Ophthalmology, Massachusetts Eye and Ear.

Publisher: Healio.com Country of Publication: United States NLM ID: 101599215 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2325-8179 (Electronic) Linking ISSN: 23258160 NLM ISO Abbreviation: Ophthalmic Surg Lasers Imaging Retina Subsets: MEDLINE

Hale AT; 1Department of Neurosurgery, University of Alabama at Birmingham, Alabama.; 2Neuroscience Institute, University of Cape Town, South Africa.; Kalailingam P; 3Center for Genomic Medicine, Massachusetts General Hospital, Boston, Massachusetts.; Kundishora AJ; 4Division of Neurosurgery, Children's Hospital of Philadelphia, Pennsylvania.; Reeves BC; 5Department of Neurosurgery, Barrow Neurological Institute, Phoenix, Arizona.; Brastianos PK; 6Department of Medicine, Massachusetts General Hospital, Boston, Massachusetts.; Shih HA; 7Department of Radiation Oncology, Massachusetts General Hospital, Boston, Massachusetts.; Jain RK; 7Department of Radiation Oncology, Massachusetts General Hospital, Boston, Massachusetts.; Ning M; 8Department of Neurology, Harvard Medical School and Massachusetts General Hospital, Boston, Massachusetts.; 9Cardio-Neurology and Clinical Proteomics Research Center, Massachusetts General Hospital, Boston, Massachusetts.; Stapleton CJ; 10Department of Neurosurgery, Harvard Medical School and Massachusetts General Hospital, Boston, Massachusetts.; Patel AB; 10Department of Neurosurgery, Harvard Medical School and Massachusetts General Hospital, Boston, Massachusetts.; Smith ER; 11Cerebrovascular Surgery and Interventions Center, Boston Children's Hospital, Boston, Massachusetts.; 12Department of Neurosurgery, Boston Children's Hospital, Boston, Massachusetts.; Chapman PH; 10Department of Neurosurgery, Harvard Medical School and Massachusetts General Hospital, Boston, Massachusetts.; Rabinov JD; 10Department of Neurosurgery, Harvard Medical School and Massachusetts General Hospital, Boston, Massachusetts.; 13Department of Radiology, Massachusetts General Hospital, Boston, Massachusetts.; Kleinstiver BP; 3Center for Genomic Medicine, Massachusetts General Hospital, Boston, Massachusetts.; 14Department of Pathology, Massachusetts General Hospital, Boston, Massachusetts.; 15Department of Pathology, Harvard Medical School, Boston, Massachusetts.; Musolino PL; 3Center for Genomic Medicine, Massachusetts General Hospital, Boston, Massachusetts.; 8Department of Neurology, Harvard Medical School and Massachusetts General Hospital, Boston, Massachusetts.; Kahle KT; 10Department of Neurosurgery, Harvard Medical School and Massachusetts General Hospital, Boston, Massachusetts.; 16Broad Institute of MIT and Harvard, Cambridge, Massachusetts.; 18Harvard Program in Neuroscience, Harvard University, Cambridge, Massachusetts.; 18Harvard Program in Neuroscience, Harvard University, Cambridge, Massachusetts.

Publisher: American Association of Neurological Surgeons Country of Publication: United States NLM ID: 0253357 Publication Model: Electronic Cited Medium: Internet ISSN: 1933-0693 (Electronic) Linking ISSN: 00223085 NLM ISO Abbreviation: J Neurosurg Subsets: MEDLINE; In Process

Tou CJ; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA. connorjtou@gmail.com.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA. connorjtou@gmail.com.; Biological Engineering Program, Massachusetts Institute of Technology, Cambridge, MA, USA. connorjtou@gmail.com.; Xie K; Full Circles Therapeutics, Cambridge, MA, USA.; Ferreira da Silva J; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Harvard Medical School, Boston, MA, USA.; Kalailingam P; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Harvard Medical School, Boston, MA, USA.; Amar-Lewis E; Institute for Medical Engineering and Science, Massachusetts Institute of Technology, Cambridge, MA, USA.; Department of Medicine, Division of Engineering in Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, MA, USA.; Wyss Institute for Biologically Inspired Engineering, Harvard University, Boston, MA, USA.; Rufino-Ramos D; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Harvard Medical School, Boston, MA, USA.; Sawyer W; Department of Medicine, Division of Engineering in Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, MA, USA.; Wyss Institute for Biologically Inspired Engineering, Harvard University, Boston, MA, USA.; Eller ML; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Starzyk J; Full Circles Therapeutics, Cambridge, MA, USA.; Majumdar I; Full Circles Therapeutics, Cambridge, MA, USA.; Wang J; Full Circles Therapeutics, Cambridge, MA, USA.; Lee D; Full Circles Therapeutics, Cambridge, MA, USA.; Yang S; Department of Bioengineering, Northeastern University, Boston, MA, USA.; Meis RJ; CELLSCRIPT, Madison, WI, USA.; Wisconsin Institute for Immune and Cell Therapy, Madison, WI, USA.; Dahl GA; CELLSCRIPT, Madison, WI, USA.; Wisconsin Institute for Immune and Cell Therapy, Madison, WI, USA.; Li J; Department of Bioengineering, Northeastern University, Boston, MA, USA.; Shan R; Full Circles Therapeutics, Cambridge, MA, USA.; Stellate DNA, Cambridge, MA, USA.; Quintara Bioscience, Cambridge, MA, USA.; Artzi N; Institute for Medical Engineering and Science, Massachusetts Institute of Technology, Cambridge, MA, USA.; Department of Medicine, Division of Engineering in Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, MA, USA.; Wyss Institute for Biologically Inspired Engineering, Harvard University, Boston, MA, USA.; Musolino PL; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Harvard Medical School, Boston, MA, USA.; Wu H; Full Circles Therapeutics, Cambridge, MA, USA. howard.wu@fullcirclestx.com.; Stellate DNA, Cambridge, MA, USA. howard.wu@fullcirclestx.com.; Kleinstiver BP; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA. bkleinstiver@mgh.harvard.edu.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA. bkleinstiver@mgh.harvard.edu.; Department of Pathology, Harvard Medical School, Boston, MA, USA. bkleinstiver@mgh.harvard.edu.

Publisher: Nature Publishing Group Country of Publication: England NLM ID: 0410462 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1476-4687 (Electronic) Linking ISSN: 00280836 NLM ISO Abbreviation: Nature Subsets: MEDLINE

Yska HAF; INSERM U 1127, CNRS UMR 7225, Sorbonne Université, UPMC Univ Paris 06 UMR S 1127, Institut du Cerveau, ICM, France.; Golse M; Department of Neuroradiology, AP-HP, Pitié-Salpêtrière University Hospital, Paris, France.; Galanaud D; Department of Neuroradiology, AP-HP, Pitié-Salpêtrière University Hospital, Paris, France.; Amartino HM; Hospital Universitario Austral, Buenos Aires, Argentina.; Bergner C; University of Leipzig Medical Center, Department of Neurology, Myelin Research Center Leipzig, Germany.; Bruschi F; COALA (Center for Diagnosis and Treatment of Leukodystrophies), Unit of Pediatric Neurology, 'Vittore Buzzi' Children's Hospital, Milan, Italy.; Department of Biomedical and Clinical Sciences 'Luigi Sacco', University of Milan, Italy.; Eichler FS; Massachusetts General Hospital, Harvard Medical School, Boston.; Fatemi A; The Moser Center for Leukodystrophies, Kennedy Krieger Institute, Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD.; García-Cazorla À; Neurometabolic Unit, Neurology Department, Institut de Recerca Sant Joan de Déu, CIBERER and MetabERN, Hospital Sant Joan de Déu, Barcelona, Spain.; Gomez-Chiari M; Diagnostic Imaging Department, Sant Joan de Déu Barcelona Children's Hospital, Esplugues de Llobregat, Spain.; Institut de Recerca Sant Joan de Déu, Sant Joan de Déu Barcelona Children's Hospital, Esplugues de Llobregat, Spain.; Köhler W; University of Leipzig Medical Center, Department of Neurology, Myelin Research Center Leipzig, Germany.; Loes D; University of Minnesota, Minneapolis.; Lund T; Division of Pediatric Blood and Marrow Transplant, University of Minnesota, Minneapolis.; Mallack EJ; The Moser Center for Leukodystrophies, Kennedy Krieger Institute, Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD.; Moscatelli M; Neuroradiology Unit, Fondazione IRCCS Istituto Neurologico C. Besta, Milano, Italy.; Musolino PL; Center for Genomic Medicine, Massachusetts General Hospital, Harvard Medical School, Boston.; Nascene DR; University of Minnesota, Minneapolis.; Orthmann-Murphy JL; Department of Neurology, University of Pennsylvania, Philadelphia.; Parazzini C; COALA (Center for Diagnosis and Treatment of Leukodystrophies), Unit of Pediatric Neurology, 'Vittore Buzzi' Children's Hospital, Milan, Italy.; Department of Pediatric Radiology and Neuroradiology, 'Vittore Buzzi' Children's Hospital, Milan, Italy.; Pouwels PJW; Department of Radiology and Nuclear Medicine, Amsterdam University Medical Center, the Netherlands.; Ribeiro-Constante J; Neurometabolic Unit, Neurology Department, Institut de Recerca Sant Joan de Déu, CIBERER and MetabERN, Hospital Sant Joan de Déu, Barcelona, Spain.; Roosendaal SD; Department of Radiology and Nuclear Medicine, Amsterdam University Medical Center, the Netherlands.; Salsano E; Unit of Rare Neurological Diseases, Fondazione IRCCS Istituto Neurologico C. Besta, Milano, Italy.; Sgobbi PV; PSEG Centro de Pesquisa Clinica, São Paulo, Brazil.; Sevin C; INSERM U 1127, CNRS UMR 7225, Sorbonne Université, UPMC Univ Paris 06 UMR S 1127, Institut du Cerveau, ICM, France.; Department of Neuropediatrics, French Reference Center for Leukodystrophies, Bicêtre Hospital, Paris, France.; Smith Fine A; The Moser Center for Leukodystrophies, Kennedy Krieger Institute, Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD.; Tonduti D; COALA (Center for Diagnosis and Treatment of Leukodystrophies), Unit of Pediatric Neurology, 'Vittore Buzzi' Children's Hospital, Milan, Italy.; Department of Biomedical and Clinical Sciences 'Luigi Sacco', University of Milan, Italy.; Van Haren K; Department of Neurology & Department of Pediatrics, Stanford University School of Medicine, CA.; Zerem A; Pediatric Neurology Institute, Dana-Dwek Children's Hospital, Tel Aviv Sourasky Medical Center, Gray Faculty of Medical and Health Sciences, Tel Aviv University, Israel.; Engelen M; Department of Child Neurology, Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Center, the Netherlands.; Amsterdam Neuroscience, University of Amsterdam, the Netherlands; and.; Mochel F; INSERM U 1127, CNRS UMR 7225, Sorbonne Université, UPMC Univ Paris 06 UMR S 1127, Institut du Cerveau, ICM, France.; AP-HP, Pitié-Salpêtrière University Hospital, Department of Medical Genetics, Reference Centers for Adult Neurometabolic Diseases and Adult Leukodystrophies, Paris, France.

Publisher: Lippincott Williams & Wilkins Country of Publication: United States NLM ID: 0401060 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1526-632X (Electronic) Linking ISSN: 00283878 NLM ISO Abbreviation: Neurology Subsets: MEDLINE

Imai T; Neurovascular Research Unit, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Charlestown, Massachusetts, USA.; Krishnan V; Center for Genomic Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Lai JH; Neurovascular Research Unit, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Charlestown, Massachusetts, USA.; Division of Neurocritical Care, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Alber E; Neurovascular Research Unit, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Charlestown, Massachusetts, USA.; Division of Neurocritical Care, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Hawley L; Neurovascular Research Unit, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Charlestown, Massachusetts, USA.; Division of Neurocritical Care, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Gandhi A; Center for Genomic Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Kalailingam P; Center for Genomic Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Yang J; Neurovascular Research Unit, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Charlestown, Massachusetts, USA.; Division of Neurocritical Care, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Tambala D; Center for Genomic Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Hike DC; Athinoula A. Martinos Center for Biomedical Imaging, Department of Radiology, Massachusetts General Hospital, Charlestown, Massachusetts, USA.; Zhou XA; Athinoula A. Martinos Center for Biomedical Imaging, Department of Radiology, Massachusetts General Hospital, Charlestown, Massachusetts, USA.; Fong C; Center for Genomic Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Ondeck B; Center for Genomic Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Cruz ETD; Center for Genomic Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Liu X; Athinoula A. Martinos Center for Biomedical Imaging, Department of Radiology, Massachusetts General Hospital, Charlestown, Massachusetts, USA.; Siv AK; Neurovascular Research Unit, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Charlestown, Massachusetts, USA.; Division of Neurocritical Care, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Öncel M; Neurovascular Research Unit, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Charlestown, Massachusetts, USA.; Division of Neurocritical Care, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Das S; Center for Genomic Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Sakadžić S; Athinoula A. Martinos Center for Biomedical Imaging, Department of Radiology, Massachusetts General Hospital, Charlestown, Massachusetts, USA.; Ayata C; Neurovascular Research Unit, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Charlestown, Massachusetts, USA.; Stroke Service, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Yu X; Athinoula A. Martinos Center for Biomedical Imaging, Department of Radiology, Massachusetts General Hospital, Charlestown, Massachusetts, USA.; Lindsay ME; Cardiovascular Genetics Program, Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Division of Pediatric Cardiology, Department of Pediatrics, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Musolino PL; Center for Genomic Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Division of Neurocritical Care, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Stroke Service, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Chung DY; Neurovascular Research Unit, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Charlestown, Massachusetts, USA.; Division of Neurocritical Care, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.

Country of Publication: United States NLM ID: 101680187 Publication Model: Electronic Cited Medium: Internet ISSN: 2692-8205 (Electronic) Linking ISSN: 26928205 NLM ISO Abbreviation: bioRxiv Subsets: PubMed not MEDLINE

Rahman AA; Department of Pediatric Surgery, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Stavely R; Department of Pediatric Surgery, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Ott LC; Department of Pediatric Surgery, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Han CY; Department of Pediatric Surgery, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Ohishi K; Department of Pediatric Surgery, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Drug Discovery Laboratory, Wakunaga Pharmaceutical Co., Ltd., Akitakata, Hiroshima, Japan.; Hotta R; Department of Pediatric Surgery, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Burns AJ; Department of Pediatric Surgery, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Das S; Center for Genomic Medicine, Massachusetts General Hospital, Boston, Massachusetts, USA.; Da Cruz E; Center for Genomic Medicine, Massachusetts General Hospital, Boston, Massachusetts, USA.; Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Tambala D; Center for Genomic Medicine, Massachusetts General Hospital, Boston, Massachusetts, USA.; Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Lindsay ME; Cardiovascular Genetics Program.; Cardiovascular Research Center, Division of Cardiology, Department of Medicine; and.; Musolino PL; Center for Genomic Medicine, Massachusetts General Hospital, Boston, Massachusetts, USA.; Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Division of Neurocritical Care, Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts, USA.; Goldstein AM; Department of Pediatric Surgery, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.

Publisher: American Society for Clinical Investigation Country of Publication: United States NLM ID: 101676073 Publication Model: eCollection Cited Medium: Internet ISSN: 2379-3708 (Electronic) Linking ISSN: 23793708 NLM ISO Abbreviation: JCI Insight Subsets: MEDLINE

Da Cruz E; Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts; Center for Genomic Medicine, Massachusetts General Hospital, Boston, Massachusetts.; Tambala D; Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts; Center for Genomic Medicine, Massachusetts General Hospital, Boston, Massachusetts.; Lynch A; Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts; Center for Genomic Medicine, Massachusetts General Hospital, Boston, Massachusetts.; Manley C; The Laboratory for Visual Neuroplasticity, Massachusetts Eye and Ear, Boston, Massachusetts.; Bambery M; Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts.; Kelly D; Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts.; Chui C; Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts.; Alhadid K; Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts; Harvard Medical School, Boston, Massachusetts.; Sullivan AW; Harvard Medical School, Boston, Massachusetts; Department of Psychiatry, Massachusetts General Hospital, Boston, Massachusetts.; Grieco J; Harvard Medical School, Boston, Massachusetts; Department of Psychiatry, Massachusetts General Hospital, Boston, Massachusetts.; Ondeck B; Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts; Center for Genomic Medicine, Massachusetts General Hospital, Boston, Massachusetts.; Lauer A; Department of Neuroradiology, Heidelberg University, Heidelberg, Germany.; Merabet LB; The Laboratory for Visual Neuroplasticity, Massachusetts Eye and Ear, Boston, Massachusetts; Harvard Medical School, Boston, Massachusetts.; Musolino PL; Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts; Center for Genomic Medicine, Massachusetts General Hospital, Boston, Massachusetts; Harvard Medical School, Boston, Massachusetts. Electronic address: pmusolino@mgh.harvard.edu.

Publisher: Elsevier Science Publishing Country of Publication: United States NLM ID: 8508183 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1873-5150 (Electronic) Linking ISSN: 08878994 NLM ISO Abbreviation: Pediatr Neurol Subsets: MEDLINE

Zhang LJ; Department of Ophthalmology, Massachusetts Eye and Ear, Harvard Medical School, Boston, Massachusetts, USA.; Hoyek S; Department of Ophthalmology, Massachusetts Eye and Ear, Harvard Medical School, Boston, Massachusetts, USA.; Lynch A; Department of Ophthalmology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Miller JB; Department of Ophthalmology, Massachusetts Eye and Ear, Harvard Medical School, Boston, Massachusetts, USA.; Gise R; Department of Ophthalmology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Musolino PL; Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Patel NA; Department of Ophthalmology, Massachusetts Eye and Ear, Harvard Medical School, Boston, Massachusetts, USA.

Publisher: Informa Healthcare Country of Publication: England NLM ID: 9436057 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1744-5094 (Electronic) Linking ISSN: 13816810 NLM ISO Abbreviation: Ophthalmic Genet Subsets: MEDLINE

Lin AE; Medical Genetics and Metabolism, Department of Pediatrics, Massachusetts General Hospital, Boston, Massachusetts, USA.; Scimone ER; Medical Genetics and Metabolism, Department of Pediatrics, Massachusetts General Hospital, Boston, Massachusetts, USA.; Thom RP; Lurie Center for Autism, Mass General for Children, Boston, Massachusetts, USA.; Department of Psychiatry, Massachusetts General Hospital, Boston, Massachusetts, USA.; Balaguru D; Pediatric Cardiology, Department of Pediatrics, Massachusetts General Hospital, Boston, Massachusetts, USA.; Kinane TB; Pediatric Pulmonary and Sleep Medicine, Department of Pediatrics, Massachusetts General Hospital, Boston, Massachusetts, USA.; Moschovis PP; Pediatric Pulmonary and Sleep Medicine, Department of Pediatrics, Massachusetts General Hospital, Boston, Massachusetts, USA.; Cohen MS; Pediatric Otorhinolaryngology, Massachusetts Eye and Ear, Boston, Massachusetts, USA.; Tan W; Pediatric Nephrology, Department of Pediatrics, Massachusetts General Hospital, Boston, Massachusetts, USA.; Hague CD; Department of Psychiatry, Massachusetts General Hospital, Boston, Massachusetts, USA.; Dannheim K; Department of Pathology, Massachusetts General Hospital, Boston, Massachusetts, USA.; Levitsky LL; Pediatric Endocrinology, Department of Pediatrics, Massachusetts General Hospital, Boston, Massachusetts, USA.; Lilly E; Department of Dermatology, Massachusetts General Hospital, Boston, Massachusetts, USA.; DiGiacomo DV; Division of Rheumatology, Allergy and Immunology, Department of Medicine, Massachusetts General Hospital, Boston, Massachusetts, USA.; Masse KM; Department of Physical Therapy, Massachusetts General Hospital, Boston, Massachusetts, USA.; Kadzielski SM; Lurie Center for Autism, Mass General for Children, Boston, Massachusetts, USA.; Pediatric Gastroenterology, Department of Pediatrics, Massachusetts General Hospital, Boston, Massachusetts, USA.; Zar-Kessler CA; Pediatric Gastroenterology, Department of Pediatrics, Massachusetts General Hospital, Boston, Massachusetts, USA.; Ginns LC; Division of Pulmonary and Critical Care Medicine, Department of Medicine, Massachusetts General Hospital, Boston, Massachusetts, USA.; Neumeyer AM; Lurie Center for Autism, Mass General for Children, Boston, Massachusetts, USA.; Pediatric Neurology, Massachusetts General Hospital, Boston, Massachusetts, USA.; Colvin MK; Department of Psychiatry, Massachusetts General Hospital, Boston, Massachusetts, USA.; Elder JS; Division of Pediatric Urology, Department of Urology, Massachusetts General Hospital, Boston, Massachusetts, USA.; Learn CP; Division of Cardiology, Department of Medicine, Corrigan Minehan Heart Center, Adult Congenital Heart Disease, Massachusetts General Hospital, Boston, Massachusetts, USA.; Mou H; Mucosal Immunology and Biology Research Center, Massachusetts General Hospital, Boston, Massachusetts, USA.; Weagle KM; Department of Child Life, Pediatric Imaging Program, Massachusetts General Hospital, Boston, Massachusetts, USA.; Buch KA; Department of Radiology, Massachusetts General Hospital, Boston, Massachusetts, USA.; Butler WE; Department of Neurosurgery, Massachusetts General Hospital, Boston, Massachusetts, USA.; Alhadid K; Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts, USA.; Musolino PL; Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts, USA.; Sultana S; Department of Radiology, Massachusetts General Hospital, Boston, Massachusetts, USA.; Bandyopadhyay D; Department of Radiology, Massachusetts General Hospital, Boston, Massachusetts, USA.; Rapalino O; Department of Radiology, Massachusetts General Hospital, Boston, Massachusetts, USA.; Peacock ZS; Oral and Maxillofacial Surgery, Massachusetts General Hospital and Harvard School of Dental Medicine, Boston, Massachusetts, USA.; Chou EL; Division of Vascular and Endovascular Surgery, Massachusetts General Hospital, Boston, Massachusetts, USA.; Heidary G; Department of Ophthalmology, Boston Children's Hospital, Boston, Massachusetts, USA.; Dorfman AT; Division of Cardiology, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.; Morris SA; Division of Cardiology, Department of Pediatrics, Texas Children's Hospital and Baylor College of Medicine, Houston, Texas, USA.; Bergin JD; Division of Cardiology, Department of Medicine, University of Virginia, Charlottesville, Virginia, USA.; Rayment JH; Respiratory Medicine, Department of Pediatrics, British Columbia Children's Hospital and University of British Columbia, Vancouver, British Columbia, Canada.; Schimmenti LA; Department of Clinical Genomics, Mayo Clinic, Rochester, Minnesota, USA.; Department of Otorhinolaryngology, Head and Neck Surgery, Mayo Clinic, Rochester, Minnesota, USA.; Department of Ophthalmology, Mayo Clinic, Rochester, Minnesota, USA.; Department of Biochemistry and Molecular Biology, Mayo Clinic, Rochester, Minnesota, USA.; Lindsay ME; Pediatric Cardiology, Department of Pediatrics, Massachusetts General Hospital, Boston, Massachusetts, USA.; Pediatric Cardiology, Department of Pediatrics, Cardiovascular Genetics Program, Cardiology Division, Department of Medicine, Massachusetts General Hospital, Boston, Massachusetts, USA.

Publisher: Wiley-Blackwell Country of Publication: United States NLM ID: 101235741 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1552-4833 (Electronic) Linking ISSN: 15524825 NLM ISO Abbreviation: Am J Med Genet A Subsets: MEDLINE

Alves CRR; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Harvard Medical School, Boston, MA, USA.; Das S; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Krishnan V; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Ha LL; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Fox LR; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Stutzman HE; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Shamber CE; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Kalailingam P; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; McCarthy S; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Lino Cardenas CL; Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Cardiovascular Research Center, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Fong CE; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Imai T; Neurovascular Research Unit, Department of Radiology, Massachusetts General Hospital, Harvard Medical School, Charlestown, MA, USA.; Mitra S; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Yun S; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Wood RK; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.; Benning FMC; Department of Molecular Biology, Massachusetts General Hospital, Boston, MA, USA.; Department of Genetics, Harvard Medical School, Boston, MA, USA.; Roh K; Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Cardiovascular Research Center, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Lawton J; Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Cardiovascular Research Center, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Kim N; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Harvard Medical School, Boston, MA, USA.; Silverstein RA; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Biological and Biomedical Sciences Program, Harvard University, Boston, MA, USA.; Ferreira da Silva J; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Harvard Medical School, Boston, MA, USA.; de la Cruz D; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Harvard Medical School, Boston, MA, USA.; Richa R; Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Cardiovascular Research Center, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Xie J; Department of Genetics and Cellular Medicine, UMass Chan Medical School, Worcester, MA, USA.; Gray-Edwards HL; Department of Genetics and Cellular Medicine, UMass Chan Medical School, Worcester, MA, USA.; Malhotra R; Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Cardiovascular Research Center, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Chung DY; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Harvard Medical School, Boston, MA, USA.; Neurovascular Research Unit, Department of Radiology, Massachusetts General Hospital, Harvard Medical School, Charlestown, MA, USA.; Division of Neurocritical Care, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Chao LH; Department of Molecular Biology, Massachusetts General Hospital, Boston, MA, USA.; Department of Genetics, Harvard Medical School, Boston, MA, USA.; Tsai SQ; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.; Maguire CA; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Harvard Medical School, Boston, MA, USA.; Lindsay ME; Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA. Lindsay.Mark@mgh.harvard.edu.; Cardiovascular Research Center, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA. Lindsay.Mark@mgh.harvard.edu.; Kleinstiver BP; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA. bkleinstiver@mgh.harvard.edu.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA. bkleinstiver@mgh.harvard.edu.; Department of Pathology, Harvard Medical School, Boston, MA, USA. bkleinstiver@mgh.harvard.edu.; Musolino PL; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA. pmusolino@mgh.harvard.edu.; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA. pmusolino@mgh.harvard.edu.; Department of Neurology, Harvard Medical School, Boston, MA, USA. pmusolino@mgh.harvard.edu.; Division of Neurocritical Care, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA. pmusolino@mgh.harvard.edu.

Publisher: Springer Nature Country of Publication: England NLM ID: 101696896 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2157-846X (Electronic) Linking ISSN: 2157846X NLM ISO Abbreviation: Nat Biomed Eng Subsets: MEDLINE

Tambala D; Department of Neurology, Massachusetts General Hospital, Boston, MA; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA.; Vassar R; Department of Neurology, University of California San Francisco, San Francisco, CA.; Snow J; Department of Neurology, Massachusetts General Hospital, Boston, MA; Harvard Medical School, Boston, MA.; Balestrini S; Neuroscience and Human Genetics Department, Meyer Children's Hospital IRCSS, Florence, Italy; University of Florence, Florence, Italy.; Bersano A; Cerebrovascular Unit Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.; Guey S; French Reference Center for Rare Vascular Diseases of the Brain and the Eye, Lariboisière Hospital, Paris, France.; Bonaventura E; Unit of Pediatric Neurology, C.O.A.L.A (Center for diagnosis and treatment of leukodystrophies), V. Buzzi Children's Hospital, Milan, Italy.; Signorini S; Developmental Neuro-Ophthalmology Unit, Child Neurology and Psychiatry Unit, IRCCS Mondino Foundation, Pavia, Italy; Child Neurology and Psychiatry Unit, IRCCS Mondino Foundation, Pavia, Italy.; Sartori S; Pediatric Neurology and Neurophysiology Unit, Department of Women's and Children's Health, University of Padua, Padua, Italy.; Bertini E; Research Unit of Neuromuscular Disease, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.; Tonduti D; Unit of Pediatric Neurology, C.O.A.L.A (Center for diagnosis and treatment of leukodystrophies), V. Buzzi Children's Hospital, Milan, Italy; Department of Biomedical and Clinical Sciences, University of Milan, Milan, Italy.; Parazzini C; Pediatric Radiology and Neuroradiology Department, Children's Hospital V. Buzzi, Milan, Italy.; Macchiaiolo M; Rare Diseases and Medical Genetics Unit, University-Hospital Pediatric Department (DPUO), Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.; Pelizza MF; Pediatric Neurology and Neurophysiology Unit, Department of Women's and Children's Health, University of Padua, Padua, Italy.; Pichiecchio A; Department of Brain and Behavioral Sciences, University of Pavia, Italy; Neuroradiology Department, IRCCS Mondino Foundation, Pavia, Italy.; Massella L; Division of Nephrology, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.; Coste T; Department of Neurovascular Genetics, AP-HP, Paris, France; Université Paris Cité, Inserm, NeuroDiterot, Paris, France.; Orcesi S; Child Neurology and Psychiatry Unit, IRCCS Mondino Foundation, Pavia, Italy; Department of Brain and Behavioral Sciences, University of Pavia, Italy.; Politano D; Child Neurology and Psychiatry Unit, IRCCS Mondino Foundation, Pavia, Italy; Department of Brain and Behavioral Sciences, University of Pavia, Italy.; Bacci G; Pediatric Ophthalmology Unit, Meyer Children's Hospital IRCCS, Florence, Italy.; Marziali E; Pediatric Ophthalmology Unit, Meyer Children's Hospital IRCCS, Florence, Italy.; Dollfus H; ERN-EYE Centre de Référence Pour les Affections Rares en Génétique Ophtalmologique (CRMR CARGO), Institut de Génétique Médicale d'Alsace (IGMA), FSMR SENSGENE, Hôpitaux Universitaires de Strasbourg, Université de Strasbourg, Strasbourg, France.; Mandelli A; Department of Pediatric Anesthesiology and Reanimation, 'Vittore Buzzi' Children's Hospital, Milan, Italy.; Chinali M; Echocardiography Laboratory of the Department of Pediatric Cardiology and Cardiac Surgery, Pediatric Hospital Bambino Gesù, Rome, Italy.; Plaisier E; Department of Nephrology, Association pour l'Utilisation du Rein Artificiel Paris Plaisance, Paris, France.; Simioni P; General Internal Medicine and Thrombotic and Hemorrhagic Unit, Department of Medicine, University Hospital of Padova, Padova, Italy.; Colombatti R; Pediatric Oncology Hematology Unit, Department of Women's and Children's Health, University of Padua, Padua, Italy.; Guerrini R; Neuroscience and Human Genetics Department, Meyer Children's Hospital IRCSS, Florence, Italy; University of Florence, Florence, Italy.; Tournier-Lasserve E; Department of Neurovascular Molecular Genetics AP-HP, Saint-Louis Hospital, Paris, France; Reference Center for Rare Vascular Diseases of the Central Nervous System and the Retina (CERVCO), APHP Lariboisière Hospital, Paris, France.; Gould DB; Departments of Ophthalmology and Anatomy, University of California San Francisco, San Francisco, CA; Cardiovascular Research Institute, University of California San Francisco, San Francisco, CA; Aging Research Institute, University of California San Francisco, San Francisco, CA; Institute for Human Genetics, University of California San Francisco, San Francisco, CA.; Musolino PL; Department of Neurology, Massachusetts General Hospital, Boston, MA; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA; Harvard Medical School, Boston, MA. Electronic address: pmusolino@mgh.harvard.edu.

Publisher: Elsevier Country of Publication: United States NLM ID: 9815831 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1530-0366 (Electronic) Linking ISSN: 10983600 NLM ISO Abbreviation: Genet Med Subsets: MEDLINE

García-Cazorla Á; Neurometabolic Unit, Neurology Department, Institut de Recerca, CIBERER and MetabERN, Hospital Sant Joan de Déu, Barcelona, Spain.; Sevin C; Pediatric Neurology Department, Assistance Publique-Hôpitaux de Paris, Hôpitaux Universitaires Paris Saclay, Bicêtre Hospital, Le Kremlin Bicêtre, Paris, France.; Constante JR; Neurometabolic Unit, Neurology Department, Institut de Recerca, CIBERER and MetabERN, Hospital Sant Joan de Déu, Barcelona, Spain.; Yazbeck E; Pediatric Neurology Department, Assistance Publique-Hôpitaux de Paris, Hôpitaux Universitaires Paris Saclay, Bicêtre Hospital, Le Kremlin Bicêtre, Paris, France.; Rosewich H; Department of Pediatrics and Adolescent Medicine, University Medical Center Göttingen, Georg August University Göttingen, Göttingen, Germany.; German Center for Child and Adolescent Health (DZKJ), Göttingen, Germany.; Department of Child Neurology, Developmental Neurology, General Pediatrics, Endocrinology, Diabetology, Social Pediatrics, University Children's Hospital, Eberhard Karls University Tübingen, Tübingen, Germany.; Jimenez S; Children's Neurodevelopment Center, Hasbro Children's Hospital, Providence, RI, USA.; Chia-Yi Chiang G; Department of Radiology, Weill Cornell Medicine, NewYork-Presbyterian Hospital, New York, NY, USA.; Rapalino O; Division of Neuroradiology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Caruso P; Pediatric Neuroimaging Service, Lenox Hill Radiology and Medical Imaging Associates, New York, NY, USA.; Balentine D; Athinoula A. Martinos Center for Biomedical Imaging, Department of Radiology, Massachusetts General Hospital, Boston, MA, USA.; Helmer KG; Athinoula A. Martinos Center for Biomedical Imaging, Department of Radiology, Massachusetts General Hospital, Boston, MA, USA.; Harvard Medical School, Boston, MA, USA.; Bennett S; CTI Clinical Trial & Consulting, Covington, KY, USA.; Emanuele M; Minoryx Therapeutics SL, Barcelona, Spain.; Rodriguez-Pascau L; Minoryx Therapeutics SL, Barcelona, Spain.; Pizcueta P; Minoryx Therapeutics SL, Barcelona, Spain.; Pina G; Minoryx Therapeutics SL, Barcelona, Spain.; Vilà A; Minoryx Therapeutics SL, Barcelona, Spain.; Rovira M; Minoryx Therapeutics SL, Barcelona, Spain.; Mantilla A; Minoryx Therapeutics SL, Barcelona, Spain.; Meya U; Minoryx Therapeutics SL, Barcelona, Spain.; Mistry A; Minoryx Therapeutics SL, Barcelona, Spain.; Pascual M; Minoryx Therapeutics SL, Barcelona, Spain.; Pascual S; Minoryx Therapeutics SL, Barcelona, Spain.; Martinell M; Minoryx Therapeutics SL, Barcelona, Spain.; Musolino PL; Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Mallack E; The Moser Center for Leukodystrophies, Kennedy Krieger Institute, Johns Hopkins University School of Medicine, Baltimore, MD, USA.

Publisher: The Lancet Country of Publication: England NLM ID: 101733727 Publication Model: eCollection Cited Medium: Internet ISSN: 2589-5370 (Electronic) Linking ISSN: 25895370 NLM ISO Abbreviation: EClinicalMedicine Subsets: PubMed not MEDLINE

Kalailingam P; Department of Neurology (P.K., P.L.M.), Massachusetts General Hospital, Boston.; Center for Genomic Medicine (P.K., P.L.M.), Massachusetts General Hospital, Boston.; Department of Neurology, Harvard Medical School, Boston, MA (P.K., P.L.M.).; Rannikmae K; Centre for Medical Informatics, Usher Institute, University of Edinburgh, United Kingdom (K.R.).; Hausman-Kedem M; Pediatric Neurology Institute, Tel Aviv Medical Center, Tel Aviv, affiliated to the Faculty of Medicine, Tel Aviv University, Israel (M.H.-K.).; Musolino PL; Department of Neurology (P.K., P.L.M.), Massachusetts General Hospital, Boston.; Center for Genomic Medicine (P.K., P.L.M.), Massachusetts General Hospital, Boston.; Department of Neurology, Harvard Medical School, Boston, MA (P.K., P.L.M.).; Ruigrok YM; Department of Neurology and Neurosurgery, University Medical Center Utrecht Brain Center, Utrecht University, the Netherlands (Y.M.R.).

Publisher: Lippincott Williams & Wilkins Country of Publication: United States NLM ID: 0235266 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1524-4628 (Electronic) Linking ISSN: 00392499 NLM ISO Abbreviation: Stroke Subsets: MEDLINE

Lauer A; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Department of Neuroradiology, Heidelberg University, Heidelberg, Germany.; Speroni SL; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Choi M; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Da X; Functional Neuroimaging Laboratory, Department of Psychiatry, Brigham and Women's Hospital, Boston, MA, USA.; Duncan C; Dana-Farber and Boston Children's Cancer and Blood Disorders Center and Harvard Medical School, Boston, MA, USA.; McCarthy S; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Krishnan V; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Lusk CA; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Rohde D; Center for Systems Biology, Massachusetts General Hospital, Boston, MA, USA.; Hansen MB; Department of Clinical Medicine, Aarhus University, Aarhus, Denmark.; Kalpathy-Cramer J; Athinoula A. Martinos Centre for Biomedical Imaging, Charlestown, MA, USA.; Loes DJ; Suburban Radiologic Consultants, Ltd, Minneapolis, MN, USA.; Caruso PA; Department of Radiology, Massachusetts General Hospital, Boston, MA, USA.; Williams DA; Dana-Farber and Boston Children's Cancer and Blood Disorders Center and Harvard Medical School, Boston, MA, USA.; Mouridsen K; Department of Clinical Medicine, Aarhus University, Aarhus, Denmark.; Emblem KE; Department of Diagnostic Physics, Oslo University Hospital, Oslo, Norway.; Eichler FS; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Musolino PL; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA. pmusolino@mgh.harvard.edu.; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA. pmusolino@mgh.harvard.edu.; Athinoula A. Martinos Centre for Biomedical Imaging, Charlestown, MA, USA. pmusolino@mgh.harvard.edu.

Publisher: Nature Pub. Group Country of Publication: England NLM ID: 101528555 Publication Model: Electronic Cited Medium: Internet ISSN: 2041-1723 (Electronic) Linking ISSN: 20411723 NLM ISO Abbreviation: Nat Commun Subsets: MEDLINE

Alves CRR; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Harvard Medical School, Boston, MA, USA.; Das S; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Krishnan V; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Ha LL; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Fox LR; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Stutzman HE; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Shamber CE; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Kalailingam P; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; McCarthy S; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Lino Cardenas CL; Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Cardiovascular Research Center, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Fong CE; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Imai T; Neurovascular Research Unit, Department of Radiology, Massachusetts General Hospital, Harvard Medical School, Charlestown, MA, USA.; Mitra S; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Yun S; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Wood RK; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.; Benning FMC; Department of Molecular Biology, Massachusetts General Hospital, Boston, MA, USA.; Department of Genetics, Harvard Medical School, Boston, MA USA.; Lawton J; Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Cardiovascular Research Center, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Kim N; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Harvard Medical School, Boston, MA, USA.; Silverstein RA; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Biological and Biomedical Sciences Program, Harvard, Boston, MA, USA.; da Silva JF; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Harvard Medical School, Boston, MA, USA.; de la Cruz D; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Harvard Medical School, Boston, MA, USA.; Richa R; Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Cardiovascular Research Center, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Malhotra R; Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Cardiovascular Research Center, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Chung DY; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Harvard Medical School, Boston, MA, USA.; Neurovascular Research Unit, Department of Radiology, Massachusetts General Hospital, Harvard Medical School, Charlestown, MA, USA.; Division of Neurocritical Care, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Chao LH; Department of Molecular Biology, Massachusetts General Hospital, Boston, MA, USA.; Department of Genetics, Harvard Medical School, Boston, MA USA.; Tsai SQ; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.; Maguire CA; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Harvard Medical School, Boston, MA, USA.; Lindsay ME; Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Cardiovascular Research Center, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Kleinstiver BP; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Harvard Medical School, Boston, MA, USA.; Musolino PL; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Harvard Medical School, Boston, MA, USA.; Division of Neurocritical Care, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.

Country of Publication: United States NLM ID: 101680187 Publication Model: Electronic Cited Medium: Internet ISSN: 2692-8205 (Electronic) Linking ISSN: 26928205 NLM ISO Abbreviation: bioRxiv Subsets: PubMed not MEDLINE

Eichler F; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.).; Duncan CN; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.).; Musolino PL; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.).; Lund TC; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.).; Gupta AO; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.).; De Oliveira S; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.).; Thrasher AJ; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.).; Aubourg P; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.).; Kühl JS; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.).; Loes DJ; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.).; Amartino H; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.).; Smith N; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.).; Folloni Fernandes J; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.).; Sevin C; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.).; Sankar R; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.).; Hussain SA; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.).; Gissen P; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.).; Dalle JH; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.).; Platzbecker U; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.).; Downey GF; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.).; McNeil E; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.).; Demopoulos L; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.).; Dietz AC; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.).; Thakar HL; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.).; Orchard PJ; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.).; Williams DA; From Massachusetts General Hospital and Harvard Medical School (F.E., P.L.M.) and Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), Boston, Bluebird Bio, Somerville (G.F.D., L.D., A.C.D., H.L.T.), and McNeil Pediatrics Consultancy, Sudbury (E.M.) - all in Massachusetts; the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (T.C.L., A.O.G., P.J.O.), and Midwest Radiology (D.J.L.) - both in Minneapolis; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (S.D.O., R.S., S.A.H.); University College London Great Ormond Street Hospital Institute of Child Health and Great Ormond Street Hospital NHS Trust, London (A.J.T., P.G.); INSERM, Université Paris-Saclay, Hôpital Kremlin-Bicêtre (P.A.), the Reference Center for Leukodystrophies, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay (C.S.), and Robert-Debre Hospital, GHU Nord-Université de Paris (J.-H.D.) - all in Paris; the Departments of Pediatric Oncology/Hematology/Hemostaseology (J.-S.K.) and Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases (U.P.), University Hospital Leipzig, Leipzig, Germany; Instituto Neurogenia and Hospital Universitario Austral - both in Buenos Aires (H.A.); Women's and Children's Health Network and the University of Adelaide - both in Adelaide, SA, Australia (N.S.); ITACI/Instituto da Criança-Hospital das Clínicas da Universidade de São Paulo, Sao Paulo (J.F.F.); and Shape Therapeutics, Seattle (A.C.D.).

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

Pacheco-Barrios K; Research Department SYNAPSIS Mental Health and Neurology Non-Profit Organization Lima Peru.; Neuromodulation Center and Center for Clinical Research Learning Spaulding Rehabilitation Hospital and Massachusetts General Hospital Boston MA USA.; Vicerrectorado de Investigación, Universidad San Ignacio de Loyola, Unidad de Investigación para la Generación y Síntesis de Evidencias en Salud Lima Peru.; Department of Epidemiology Harvard T.H. Chan School of Public Health Boston MA.; Giannoni-Luza S; Research Department SYNAPSIS Mental Health and Neurology Non-Profit Organization Lima Peru.; Neuromodulation Center and Center for Clinical Research Learning Spaulding Rehabilitation Hospital and Massachusetts General Hospital Boston MA USA.; Navarro-Flores A; Research Department SYNAPSIS Mental Health and Neurology Non-Profit Organization Lima Peru.; International Max Planck Research School for Neurosciences, Georg-August-University Göttingen Göttingen Germany.; Rebello-Sanchez I; Neuromodulation Center and Center for Clinical Research Learning Spaulding Rehabilitation Hospital and Massachusetts General Hospital Boston MA USA.; Parente J; Neuromodulation Center and Center for Clinical Research Learning Spaulding Rehabilitation Hospital and Massachusetts General Hospital Boston MA USA.; Balbuena A; Neuromodulation Center and Center for Clinical Research Learning Spaulding Rehabilitation Hospital and Massachusetts General Hospital Boston MA USA.; de Melo PS; Neuromodulation Center and Center for Clinical Research Learning Spaulding Rehabilitation Hospital and Massachusetts General Hospital Boston MA USA.; Otiniano-Sifuentes R; Departamento de Enfermedades Neurovasculares Instituto Nacional de Ciencias Neurológicas Lima Peru.; Rivera-Torrejón O; Facultad de Medicina Universidad Nacional Mayor de San Marcos Lima Peru.; Servicio de Neurología, Departamento de Medicina y Oficina de Apoyo a la Docencia e Investigación (OADI) Hospital Daniel Alcides Carrión Callao Peru.; Red de Eficacia Clínica y Sanitaria, REDECS Lima Peru.; Abanto C; Departamento de Enfermedades Neurovasculares Instituto Nacional de Ciencias Neurológicas Lima Peru.; Alva-Diaz C; Servicio de Neurología, Departamento de Medicina y Oficina de Apoyo a la Docencia e Investigación (OADI) Hospital Daniel Alcides Carrión Callao Peru.; Red de Eficacia Clínica y Sanitaria, REDECS Lima Peru.; Universidad Señor de Sipán Chiclayo Peru.; Musolino PL; Department of Neurology Massachusetts General Hospital, Harvard Medical School Boston MA.; Center for Genomic Medicine, Center for Rare Neurological Disorders Massachusetts General Hospital, Harvard Medical School Boston.; Fregni F; Neuromodulation Center and Center for Clinical Research Learning Spaulding Rehabilitation Hospital and Massachusetts General Hospital Boston MA USA.; Department of Epidemiology Harvard T.H. Chan School of Public Health Boston MA.

Publisher: Wiley-Blackwell Country of Publication: England NLM ID: 101580524 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2047-9980 (Electronic) Linking ISSN: 20479980 NLM ISO Abbreviation: J Am Heart Assoc Subsets: MEDLINE

Rost, Natalia S; Cougo, Pedro; Lorenzano, Svetlana; Li, Hua; Cloonan, Lisa; Bouts, Mark JRJ; Lauer, Arne; Etherton, Mark R; Karadeli, Hasan H; Musolino, Patricia L; Copen, William A; Arai, Ken; Lo, Eng H; Feske, Steve K; Furie, Karen L; Wu, Ona

Journal of Cerebral Blood Flow & Metabolism. Jan 01, 2018 38(1):75-86

Lauer, Arne; Da, Xiao; Hansen, Mikkel Bo; Boulouis, Gregoire; Ou, Yangming; Cai, Xuezhu; Liberato Celso Pedrotti, Afonso; Kalpathy-Cramer, Jayashree; Caruso, Paul; Hayden, Douglas L; Rost, Natalia; Mouridsen, Kim; Eichler, Florian S; Rosen, Bruce; Musolino, Patricia L

Brain. Dec 01, 2017 140(12):3139-3152