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Optimization of systemic AAV9 gene therapy in Niemann-Pick disease, type C1 mice.

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Title: Optimization of systemic AAV9 gene therapy in Niemann-Pick disease, type C1 mice.
Authors: Mylvara AV; Department of Human Health and Services, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD, USA.; Department of Human Health and Services, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA., Gibson AL; Department of Human Health and Services, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.; Howard Hughes Medical Institute, Department of Cellular and Molecular Medicine, Section of Neurobiology, Division of Biological Sciences, University of California San Diego, San Diego, CA, USA., Gu T; Department of Human Health and Services, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.; University of North Carolina, Chapel Hill, NC, USA., Davidson CD; Department of Human Health and Services, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD, USA cristin.davidson@nih.gov.; Department of Human Health and Services, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA., Incao AA; Department of Human Health and Services, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA., Melnyk K; Department of Human Health and Services, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD, USA., Gembic SR; Department of Human Health and Services, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD, USA., Pierre-Jacques D; University of Illinois Chicago, Chicago, IL, USA., Cologna SM; University of Illinois Chicago, Chicago, IL, USA., Venditti CP; Department of Human Health and Services, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA., Porter FD; Department of Human Health and Services, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD, USA fdporter@mail.nih.gov., Pavan WJ; Department of Human Health and Services, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.
Source: Life science alliance [Life Sci Alliance] 2026 Mar 30; Vol. 9 (6). Date of Electronic Publication: 2026 Mar 30 (Print Publication: 2026).
Publication Type: Journal Article
Journal Info: Publisher: Life Science Alliance, LLC Country of Publication: United States NLM ID: 101728869 Publication Model: Electronic-Print Cited Medium: Internet ISSN: 2575-1077 (Electronic) Linking ISSN: 25751077 NLM ISO Abbreviation: Life Sci Alliance Subsets: MEDLINE
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  Data: <searchLink fieldCode="AU" term="%22Mylvara+AV%22">Mylvara AV</searchLink>; Department of Human Health and Services, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD, USA.; Department of Human Health and Services, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Gibson+AL%22">Gibson AL</searchLink>; Department of Human Health and Services, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.; Howard Hughes Medical Institute, Department of Cellular and Molecular Medicine, Section of Neurobiology, Division of Biological Sciences, University of California San Diego, San Diego, CA, USA.<br /><searchLink fieldCode="AU" term="%22Gu+T%22">Gu T</searchLink>; Department of Human Health and Services, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.; University of North Carolina, Chapel Hill, NC, USA.<br /><searchLink fieldCode="AU" term="%22Davidson+CD%22">Davidson CD</searchLink>; Department of Human Health and Services, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD, USA cristin.davidson@nih.gov.; Department of Human Health and Services, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Incao+AA%22">Incao AA</searchLink>; Department of Human Health and Services, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Melnyk+K%22">Melnyk K</searchLink>; Department of Human Health and Services, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Gembic+SR%22">Gembic SR</searchLink>; Department of Human Health and Services, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Pierre-Jacques+D%22">Pierre-Jacques D</searchLink>; University of Illinois Chicago, Chicago, IL, USA.<br /><searchLink fieldCode="AU" term="%22Cologna+SM%22">Cologna SM</searchLink>; University of Illinois Chicago, Chicago, IL, USA.<br /><searchLink fieldCode="AU" term="%22Venditti+CP%22">Venditti CP</searchLink>; Department of Human Health and Services, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Porter+FD%22">Porter FD</searchLink>; Department of Human Health and Services, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD, USA fdporter@mail.nih.gov.<br /><searchLink fieldCode="AU" term="%22Pavan+WJ%22">Pavan WJ</searchLink>; Department of Human Health and Services, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.
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