In vivo haemopoietic stem cell gene therapy enabled by postnatal trafficking.

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Bibliographic Details
Title: In vivo haemopoietic stem cell gene therapy enabled by postnatal trafficking.
Authors: Milani M; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy. milani.michela@hsr.it., Fabiano A; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Perez-Rodriguez M; Biomedical Innovation Unit, Center for Research on Energy, Environment and Technology (CIEMAT), Madrid, Spain.; Biomedical Network Research Center for Rare Diseases (CIBERER), Madrid, Spain.; Sanitary Research Institute Fundación Jiménez Díaz (U.A.M), Madrid, Spain., Hernandez RJ; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Zecchillo A; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Translational and Molecular Medicine (DIMET), University of Milano Bicocca, Milan, Italy., Zonari E; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Ottonello S; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Vita-Salute San Raffaele University, Milan, Italy., Basso-Ricci L; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Canepari C; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Volpin M; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Iannello V; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Vita-Salute San Raffaele University, Milan, Italy., Capo V; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy., Quaranta P; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Vita-Salute San Raffaele University, Milan, Italy., Seffin L; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Vita-Salute San Raffaele University, Milan, Italy., Russo F; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Biffi M; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Ormoli L; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Brombin C; University Center for Statistics in the Biomedical Sciences, Vita-Salute San Raffaele University, Milan, Italy., Carlucci F; Department of Medical Biotechnologies, University of Siena, Siena, Italy., Forlino A; Department of Molecular Medicine, Biochemistry Unit, University of Pavia, Pavia, Italy., Filibian M; Biomedical Imaging Laboratory, Centro Grandi Strumenti, University of Pavia, Pavia, Italy., Montini E; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Scala S; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Villa A; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy., Bueren JA; Biomedical Innovation Unit, Center for Research on Energy, Environment and Technology (CIEMAT), Madrid, Spain.; Biomedical Network Research Center for Rare Diseases (CIBERER), Madrid, Spain.; Sanitary Research Institute Fundación Jiménez Díaz (U.A.M), Madrid, Spain., Rio P; Biomedical Innovation Unit, Center for Research on Energy, Environment and Technology (CIEMAT), Madrid, Spain.; Biomedical Network Research Center for Rare Diseases (CIBERER), Madrid, Spain.; Sanitary Research Institute Fundación Jiménez Díaz (U.A.M), Madrid, Spain., Aiuti A; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Vita-Salute San Raffaele University, Milan, Italy., Cantore A; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Vita-Salute San Raffaele University, Milan, Italy., Naldini L; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy. naldini.luigi@hsr.it.; Vita-Salute San Raffaele University, Milan, Italy. naldini.luigi@hsr.it.
Source: Nature [Nature] 2025 Jul; Vol. 643 (8073), pp. 1097-1106. Date of Electronic Publication: 2025 May 28.
Publication Type: Journal Article; Research Support, Non-U.S. Gov't
Journal Info: 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
Database: MEDLINE Ultimate
Description
ISSN:1476-4687
DOI:10.1038/s41586-025-09070-3