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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.
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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  Data: Liver-directed lentiviral gene therapy confers durable hepatic and systemic amelioration of methylmalonic acidemia in mice.
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  Data: <searchLink fieldCode="AU" term="%22Barbon+E%22">Barbon E</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Simoni+C%22">Simoni C</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="%22Argabright+A%22">Argabright A</searchLink>; Department of Biochemistry and Molecular Genetics, University of Colorado Anschutz Medical Campus, Aurora, CO, USA.<br /><searchLink fieldCode="AU" term="%22Boettiger+M%22">Boettiger M</searchLink>; Department of Biochemistry and Molecular Genetics, University of Colorado Anschutz Medical Campus, Aurora, CO, USA.<br /><searchLink fieldCode="AU" term="%22Negri+C%22">Negri C</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Manta+E%22">Manta E</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Raimondi+A%22">Raimondi A</searchLink>; ALEMBIC Advanced Light and Electron Microscopy BioImaging Center, IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Vezzoli+E%22">Vezzoli E</searchLink>; Center for Statistics in the Biomedical Sciences, Vita-Salute San Raffaele University, 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="%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="%22Nonis+A%22">Nonis A</searchLink>; Newborn Screening, Clinical Biochemistry and Clinical Pharmacy Lab Meyer Children's Hospital IRCCS, Florence, Italy.<br /><searchLink fieldCode="AU" term="%22Gazzo+F%22">Gazzo F</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Benedicenti+F%22">Benedicenti F</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, 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="%22la+Marca+G%22">la Marca G</searchLink>; Department of Experimental and Clinical Biomedical Sciences, University of Florence, Italy; IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Sanvito+F%22">Sanvito F</searchLink>; ALEMBIC Advanced Light and Electron Microscopy BioImaging Center, IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Smith+J%22">Smith J</searchLink>; Genespire S.r.l., Milan, Italy.<br /><searchLink fieldCode="AU" term="%22D'Alessandro+A%22">D'Alessandro A</searchLink>; Department of Biochemistry and Molecular Genetics, University of Colorado Anschutz Medical Campus, Aurora, CO, USA.<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. Electronic address: cantore.alessio@hsr.it.
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