Correction of osteopetrosis in the neonate oc/oc murine model after lentiviral vector gene therapy and non-genotoxic conditioning.

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Title: Correction of osteopetrosis in the neonate oc/oc murine model after lentiviral vector gene therapy and non-genotoxic conditioning.
Authors: Penna S; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy., Zecchillo A; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.; Translational and Molecular Medicine (DIMET), University of Milano Bicocca, Milan, Italy., Di Verniere M; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy., Fontana E; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy.; Humanitas Research Hospital IRCCS, Rozzano, MI, Italy., Iannello V; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.; Vita-Salute San Raffaele University, Milan, Italy., Palagano E; Humanitas Research Hospital IRCCS, Rozzano, MI, Italy.; Florence Unit, Istituto di Bioscienze e Biorisorse, Consiglio Nazionale delle Ricerche, Sesto Fiorentino, Italy., Mantero S; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy.; Humanitas Research Hospital IRCCS, Rozzano, MI, Italy., Cappelleri A; Mouse and Animal Pathology Laboratory, UniMi Foundation, Milan, Italy.; Department of Veterinary Medicine and Animal Sciences, University of Milan, Lodi, Italy., Rizzoli E; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.; Vita-Salute San Raffaele University, Milan, Italy., Santi L; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy., Crisafulli L; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy.; Humanitas Research Hospital IRCCS, Rozzano, MI, Italy., Filibian M; Biomedical Imaging Laboratory, Centro Grandi Strumenti, University of Pavia, Pavia, Italy., Forlino A; Department of Molecular Medicine, Biochemistry Unit, University of Pavia, Pavia, Italy., Basso-Ricci L; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy., Scala S; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy., Scanziani E; Mouse and Animal Pathology Laboratory, UniMi Foundation, Milan, Italy.; Department of Veterinary Medicine and Animal Sciences, University of Milan, Lodi, Italy., Schinke T; Department of Osteology and Biomechanics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany., Ficara F; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy.; Humanitas Research Hospital IRCCS, Rozzano, MI, Italy., Sobacchi C; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy.; Humanitas Research Hospital IRCCS, Rozzano, MI, Italy., Villa A; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy., Capo V; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy.
Source: Frontiers in endocrinology [Front Endocrinol (Lausanne)] 2024 Sep 09; Vol. 15, pp. 1450349. Date of Electronic Publication: 2024 Sep 09 (Print Publication: 2024).
Publication Type: Journal Article
Journal Info: Publisher: Frontiers Research Foundation] Country of Publication: Switzerland NLM ID: 101555782 Publication Model: eCollection Cited Medium: Print ISSN: 1664-2392 (Print) Linking ISSN: 16642392 NLM ISO Abbreviation: Front Endocrinol (Lausanne) Subsets: MEDLINE; PubMed not MEDLINE
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  Data: Correction of osteopetrosis in the neonate oc/oc murine model after lentiviral vector gene therapy and non-genotoxic conditioning.
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  Data: <searchLink fieldCode="AU" term="%22Penna+S%22">Penna S</searchLink>; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), 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 (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.; Translational and Molecular Medicine (DIMET), University of Milano Bicocca, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Di+Verniere+M%22">Di Verniere M</searchLink>; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Fontana+E%22">Fontana E</searchLink>; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy.; Humanitas Research Hospital IRCCS, Rozzano, MI, Italy.<br /><searchLink fieldCode="AU" term="%22Iannello+V%22">Iannello V</searchLink>; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.; Vita-Salute San Raffaele University, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Palagano+E%22">Palagano E</searchLink>; Humanitas Research Hospital IRCCS, Rozzano, MI, Italy.; Florence Unit, Istituto di Bioscienze e Biorisorse, Consiglio Nazionale delle Ricerche, Sesto Fiorentino, Italy.<br /><searchLink fieldCode="AU" term="%22Mantero+S%22">Mantero S</searchLink>; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy.; Humanitas Research Hospital IRCCS, Rozzano, MI, Italy.<br /><searchLink fieldCode="AU" term="%22Cappelleri+A%22">Cappelleri A</searchLink>; Mouse and Animal Pathology Laboratory, UniMi Foundation, Milan, Italy.; Department of Veterinary Medicine and Animal Sciences, University of Milan, Lodi, Italy.<br /><searchLink fieldCode="AU" term="%22Rizzoli+E%22">Rizzoli E</searchLink>; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.; Vita-Salute San Raffaele University, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Santi+L%22">Santi L</searchLink>; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Crisafulli+L%22">Crisafulli L</searchLink>; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy.; Humanitas Research Hospital IRCCS, Rozzano, MI, 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="%22Forlino+A%22">Forlino A</searchLink>; Department of Molecular Medicine, Biochemistry Unit, University of Pavia, Pavia, Italy.<br /><searchLink fieldCode="AU" term="%22Basso-Ricci+L%22">Basso-Ricci L</searchLink>; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), 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 (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Scanziani+E%22">Scanziani E</searchLink>; Mouse and Animal Pathology Laboratory, UniMi Foundation, Milan, Italy.; Department of Veterinary Medicine and Animal Sciences, University of Milan, Lodi, Italy.<br /><searchLink fieldCode="AU" term="%22Schinke+T%22">Schinke T</searchLink>; Department of Osteology and Biomechanics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.<br /><searchLink fieldCode="AU" term="%22Ficara+F%22">Ficara F</searchLink>; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy.; Humanitas Research Hospital IRCCS, Rozzano, MI, Italy.<br /><searchLink fieldCode="AU" term="%22Sobacchi+C%22">Sobacchi C</searchLink>; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy.; Humanitas Research Hospital IRCCS, Rozzano, MI, Italy.<br /><searchLink fieldCode="AU" term="%22Villa+A%22">Villa A</searchLink>; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), 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="%22Capo+V%22">Capo V</searchLink>; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy.
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  Data: <searchLink fieldCode="JN" term="%22101555782%22">Frontiers in endocrinology</searchLink> [Front Endocrinol (Lausanne)] 2024 Sep 09; Vol. 15, pp. 1450349. <i>Date of Electronic Publication: </i>2024 Sep 09 (<i>Print Publication: </i>2024).
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Frontiers+Research+Foundation]%22">Frontiers Research Foundation] </searchLink><i>Country of Publication: </i>Switzerland <i>NLM ID: </i>101555782 <i>Publication Model: </i>eCollection <i>Cited Medium: </i>Print <i>ISSN: </i>1664-2392 (Print) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2216642392%22">16642392 </searchLink><i>NLM ISO Abbreviation: </i>Front Endocrinol (Lausanne) <i>Subsets: </i>MEDLINE; PubMed not MEDLINE
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