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Neonatal gene therapy effectively prevents disease manifestations in a murine model of Mucopolysaccharidosis type I.

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Title: Neonatal gene therapy effectively prevents disease manifestations in a murine model of Mucopolysaccharidosis type I.
Authors: De Ponti G; San Raffaele Telethon Institute for Gene Therapy - SR-Tiget, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.; Tettamanti Center, Fondazione IRCCS San Gerardo dei Tintori, 20900 Monza, Italy., Santi L; San Raffaele Telethon Institute for Gene Therapy - SR-Tiget, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy., Dina G; Experimental Neuropathology Unit, INSpe, Division of Neuroscience, IRCCS Ospedale San Raffaele, 20132 Milan, Italy., Pievani A; Tettamanti Center, Fondazione IRCCS San Gerardo dei Tintori, 20900 Monza, Italy., Donsante S; Tettamanti Center, Fondazione IRCCS San Gerardo dei Tintori, 20900 Monza, Italy., Riminucci M; Department of Molecular Medicine, Sapienza University, 00161 Rome, Italy., Corsi A; Department of Molecular Medicine, Sapienza University, 00161 Rome, Italy., Khan S; Department of Biomedical Research, Alfred I. duPont Hospital for Children, Wilmington, DE 19803, USA., Passerini L; San Raffaele Telethon Institute for Gene Therapy - SR-Tiget, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy., Annoni A; San Raffaele Telethon Institute for Gene Therapy - SR-Tiget, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy., Gregori S; San Raffaele Telethon Institute for Gene Therapy - SR-Tiget, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy., Crippa S; San Raffaele Telethon Institute for Gene Therapy - SR-Tiget, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy., Biondi A; Pediatrics, Fondazione IRCCS San Gerardo dei Tintori, 20900 Monza, Italy.; School of Medicine and Surgery, University of Milano-Bicocca, 20900 Monza, Italy., Quattrini A; Experimental Neuropathology Unit, INSpe, Division of Neuroscience, IRCCS Ospedale San Raffaele, 20132 Milan, Italy., Tomatsu S; Department of Biomedical Research, Alfred I. duPont Hospital for Children, Wilmington, DE 19803, USA., Aiuti A; San Raffaele Telethon Institute for Gene Therapy - SR-Tiget, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.; Vita-Salute San Raffaele University, 20132 Milan, Italy.; Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, 20132 Milan, Italy., Bernardo ME; San Raffaele Telethon Institute for Gene Therapy - SR-Tiget, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.; Vita-Salute San Raffaele University, 20132 Milan, Italy.; Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, 20132 Milan, Italy., Serafini M; Tettamanti Center, Fondazione IRCCS San Gerardo dei Tintori, 20900 Monza, Italy.; School of Medicine and Surgery, University of Milano-Bicocca, 20900 Monza, Italy.
Source: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2025 Jul 30; Vol. 33 (3), pp. 101544. Date of Electronic Publication: 2025 Jul 30 (Print Publication: 2025).
Publication Type: Journal Article
Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 101624857 Publication Model: eCollection Cited Medium: Print ISSN: 2329-0501 (Print) Linking ISSN: 23290501 NLM ISO Abbreviation: Mol Ther Methods Clin Dev Subsets: PubMed not MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:2329-0501
DOI:10.1016/j.omtm.2025.101544