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

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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
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  Data: In vivo haemopoietic stem cell gene therapy enabled by postnatal trafficking.
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  Data: <searchLink fieldCode="AU" term="%22Milani+M%22">Milani M</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy. milani.michela@hsr.it.<br /><searchLink fieldCode="AU" term="%22Fabiano+A%22">Fabiano A</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Perez-Rodriguez+M%22">Perez-Rodriguez M</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Hernandez+RJ%22">Hernandez RJ</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Zecchillo+A%22">Zecchillo A</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Zonari+E%22">Zonari E</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Ottonello+S%22">Ottonello S</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Vita-Salute San Raffaele University, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Basso-Ricci+L%22">Basso-Ricci L</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Canepari+C%22">Canepari C</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Volpin+M%22">Volpin M</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Iannello+V%22">Iannello V</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Vita-Salute San Raffaele University, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Capo+V%22">Capo V</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Quaranta+P%22">Quaranta P</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Vita-Salute San Raffaele University, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Seffin+L%22">Seffin L</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Vita-Salute San Raffaele University, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Russo+F%22">Russo F</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Biffi+M%22">Biffi M</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Ormoli+L%22">Ormoli L</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Brombin+C%22">Brombin C</searchLink>; University Center for Statistics in the Biomedical Sciences, Vita-Salute San Raffaele University, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Carlucci+F%22">Carlucci F</searchLink>; Department of Medical Biotechnologies, University of Siena, Siena, Italy.<br /><searchLink fieldCode="AU" term="%22Forlino+A%22">Forlino A</searchLink>; Department of Molecular Medicine, Biochemistry Unit, University of Pavia, Pavia, Italy.<br /><searchLink fieldCode="AU" term="%22Filibian+M%22">Filibian M</searchLink>; Biomedical Imaging Laboratory, Centro Grandi Strumenti, University of Pavia, Pavia, Italy.<br /><searchLink fieldCode="AU" term="%22Montini+E%22">Montini E</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Scala+S%22">Scala S</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Villa+A%22">Villa A</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Bueren+JA%22">Bueren JA</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Rio+P%22">Rio P</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Aiuti+A%22">Aiuti A</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Vita-Salute San Raffaele University, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Cantore+A%22">Cantore A</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Vita-Salute San Raffaele University, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Naldini+L%22">Naldini L</searchLink>; 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.
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