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Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome

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source: The New England journal of medicine

year: 2021

authors: Gentner B,Tucci F,Galimberti S,Fumagalli F,De Pellegrin M,Silvani P,Camesasca C,Pontesilli S,Darin S,Ciotti F,Sarzana M,Consiglieri G,Filisetti C,Forni G,Passerini L,Tomasoni D,Cesana D,Calabria A,Spinozzi G,Cicalese MP,Calbi V,Migliavacca M,Barzaghi F,Ferrua F,Gallo V,Miglietta S,Zonari E,Cheruku PS,Forni C,Facchini M,Corti A,Gabaldo M,Zancan S,Gasperini S,Rovelli A,Boelens JJ,Jones SA,Wynn R,Baldoli C,Montini E,Gregori S,Ciceri F,Valsecchi MG,la Marca G,Parini R,Naldini L,Aiuti A,Bernardo ME,

summary/abstract:

Allogeneic hematopoietic stem-cell transplantation is the standard of care for Hurler syndrome (mucopolysaccharidosis type I, Hurler variant [MPSIH]). However, this treatment is only partially curative and is associated with complications.

organization: From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

DOI: 10.1056/NEJMoa2106596

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