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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

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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
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Volume 4, No. 10
October 2021
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  • Neuroscience
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