Liver-directed lentiviral gene therapy confers durable hepatic and systemic amelioration of methylmalonic acidemia in mice.
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| Title: | Liver-directed lentiviral gene therapy confers durable hepatic and systemic amelioration of methylmalonic acidemia in mice. |
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| Authors: | Barbon E; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Simoni C; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy; Vita-Salute San Raffaele University, Milan, Italy., Argabright A; Department of Biochemistry and Molecular Genetics, University of Colorado Anschutz Medical Campus, Aurora, CO, USA., Boettiger M; Department of Biochemistry and Molecular Genetics, University of Colorado Anschutz Medical Campus, Aurora, CO, USA., Negri C; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Manta E; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Raimondi A; ALEMBIC Advanced Light and Electron Microscopy BioImaging Center, IRCCS San Raffaele Scientific Institute, Milan, Italy., Vezzoli E; Center for Statistics in the Biomedical Sciences, Vita-Salute San Raffaele University, Milan, Italy., Canepari C; 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., Nonis A; Newborn Screening, Clinical Biochemistry and Clinical Pharmacy Lab Meyer Children's Hospital IRCCS, Florence, Italy., Gazzo F; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Benedicenti F; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Montini E; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., la Marca G; Department of Experimental and Clinical Biomedical Sciences, University of Florence, Italy; IRCCS San Raffaele Scientific Institute, Milan, Italy., Sanvito F; ALEMBIC Advanced Light and Electron Microscopy BioImaging Center, IRCCS San Raffaele Scientific Institute, Milan, Italy., Smith J; Genespire S.r.l., Milan, Italy., D'Alessandro A; Department of Biochemistry and Molecular Genetics, University of Colorado Anschutz Medical Campus, Aurora, CO, USA., Cantore A; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy; Vita-Salute San Raffaele University, Milan, Italy. Electronic address: cantore.alessio@hsr.it. |
| Source: | Journal of hepatology [J Hepatol] 2026 May 25. Date of Electronic Publication: 2026 May 25. |
| Publication Type: | Journal Article |
| Journal Info: | Publisher: Elsevier Country of Publication: Netherlands NLM ID: 8503886 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1600-0641 (Electronic) Linking ISSN: 01688278 NLM ISO Abbreviation: J Hepatol Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
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