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
Database: MEDLINE Ultimate
Description
ISSN:1664-2392
DOI:10.3389/fendo.2024.1450349