Improved systemic AAV gene therapy with a neurotrophic capsid in Niemann–Pick disease type C1 mice

Cristin D Davidson; Alana L Gibson; Tansy Gu; Laura L Baxter; Benjamin E Deverman; Keith Beadle; Arturo A Incao; Jorge L Rodriguez-Gil; Hideji Fujiwara; Xuntian Jiang; Randy J Chandler; Daniel S Ory; Viviana Gradinaru; Charles P Venditti; William J Pavan

doi:10.26508/lsa.202101040

Research Article

Open Access

Improved systemic AAV gene therapy with a neurotrophic capsid in Niemann–Pick disease type C1 mice

View ORCID ProfileCristin D Davidson, View ORCID ProfileAlana L Gibson, Tansy Gu, Laura L Baxter, Benjamin E Deverman, View ORCID ProfileKeith Beadle, Arturo A Incao, View ORCID ProfileJorge L Rodriguez-Gil, Hideji Fujiwara, Xuntian Jiang, View ORCID ProfileRandy J Chandler, Daniel S Ory, Viviana Gradinaru, View ORCID ProfileCharles P Venditti Correspondence email, View ORCID ProfileWilliam J Pavan Correspondence email

Cristin D Davidson

1Genetic Disease Research Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA

Roles: Conceptualization, Formal analysis, Investigation, Visualization, Project administration, Writing—original draft, review, and editing

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ORCID record for Cristin D Davidson

Alana L Gibson

1Genetic Disease Research Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA

Roles: Investigation, Writing—original draft, review, and editing

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ORCID record for Alana L Gibson

Tansy Gu

1Genetic Disease Research Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA

Roles: Investigation, Writing—original draft, review, and editing

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Laura L Baxter

1Genetic Disease Research Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA

Roles: Formal analysis, Visualization, Writing—original draft, review, and editing

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Benjamin E Deverman

2Division of Biology and Biological Engineering, California Institutes of Technology, Pasadena, CA, USA

Roles: Conceptualization, Resources, Investigation, Writing—review and editing

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Keith Beadle

2Division of Biology and Biological Engineering, California Institutes of Technology, Pasadena, CA, USA

Roles: Investigation, Writing—review and editing

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ORCID record for Keith Beadle

Arturo A Incao

1Genetic Disease Research Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA

Roles: Investigation, Writing—review and editing

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Jorge L Rodriguez-Gil

1Genetic Disease Research Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA

Roles: Formal analysis, Investigation, Writing—review and editing

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ORCID record for Jorge L Rodriguez-Gil

Hideji Fujiwara

3Department of Medicine, Washington University School of Medicine, St. Louis, MO, USA

Roles: Formal analysis, Writing—review and editing

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Xuntian Jiang

3Department of Medicine, Washington University School of Medicine, St. Louis, MO, USA

Roles: Formal analysis, Investigation, Writing—review and editing

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Randy J Chandler

4Medical Genomics and Metabolic Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA

Roles: Conceptualization, Writing—review and editing

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ORCID record for Randy J Chandler

Daniel S Ory

3Department of Medicine, Washington University School of Medicine, St. Louis, MO, USA

Roles: Resources, Writing—review and editing

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Viviana Gradinaru

2Division of Biology and Biological Engineering, California Institutes of Technology, Pasadena, CA, USA

Roles: Resources, Funding acquisition, Writing—review and editing

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Charles P Venditti

4Medical Genomics and Metabolic Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA

Roles: Conceptualization, Resources, Supervision, Funding acquisition, Writing—review and editing

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ORCID record for Charles P Venditti
For correspondence: venditti@mail.nih.gov

William J Pavan

1Genetic Disease Research Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA

Roles: Conceptualization, Resources, Supervision, Funding acquisition, Project administration, Writing—review and editing

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ORCID record for William J Pavan
For correspondence: bpavan@mail.nih.gov

Published 18 August 2021. DOI: 10.26508/lsa.202101040

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Neurotrophic AAV to treat NPC disease

Cristin D Davidson, Alana L Gibson, Tansy Gu, Laura L Baxter, Benjamin E Deverman, Keith Beadle, Arturo A Incao, Jorge L Rodriguez-Gil, Hideji Fujiwara, Xuntian Jiang, Randy J Chandler, Daniel S Ory, Viviana Gradinaru, Charles P Venditti, William J Pavan

Life Science Alliance Aug 2021, 4 (10) e202101040; DOI: 10.26508/lsa.202101040