Comparison of neonatal systemic and intracerebroventricular AAV9 gene therapy delivery demonstrating improved behavioral and phenotypic outcomes in a mouse model of Niemann-Pick disease, type C1

• Benjamin E. Epstein,

  Roles Conceptualization, Data curation, Formal analysis, Investigation, Methodology, Visualization, Writing – original draft, Writing – review & editing

  Affiliation Genomics, Development and Disease Section, Computational and Statistical Genomics Branch, National Human Genome Research Institute (NHGRI), National Institutes of Health, Bethesda, Maryland, United States of America

  https://orcid.org/0000-0002-5129-9044

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• Gabrielle M. Soden,

  Roles Data curation, Investigation, Writing – review & editing

  Affiliation Genomics, Development and Disease Section, Computational and Statistical Genomics Branch, National Human Genome Research Institute (NHGRI), National Institutes of Health, Bethesda, Maryland, United States of America

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• Arturo A. Incao,

  Roles Investigation, Methodology

  Affiliation Genomics, Development and Disease Section, Computational and Statistical Genomics Branch, National Human Genome Research Institute (NHGRI), National Institutes of Health, Bethesda, Maryland, United States of America

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• Avani Mylvara,

  Roles Data curation, Investigation, Visualization, Writing – review & editing

  Affiliation Genomics, Development and Disease Section, Computational and Statistical Genomics Branch, National Human Genome Research Institute (NHGRI), National Institutes of Health, Bethesda, Maryland, United States of America

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• Jonathan Flynn,

  Roles Conceptualization, Investigation, Resources, Writing – review & editing

  Affiliation Biologics Research Center, Novartis, San Diego, California, United States of America

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• Fatih Ozsolak,

  Roles Conceptualization, Funding acquisition, Project administration, Resources, Supervision, Writing – review & editing

  Affiliation Biologics Research Center, Novartis, San Diego, California, United States of America

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• William J. Pavan

  Roles Conceptualization, Funding acquisition, Project administration, Resources, Supervision, Writing – review & editing

  * E-mail: wjpavan@gmail.com

  Affiliation Genomics, Development and Disease Section, Computational and Statistical Genomics Branch, National Human Genome Research Institute (NHGRI), National Institutes of Health, Bethesda, Maryland, United States of America

  https://orcid.org/0000-0001-8281-5120

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