Structural characterization of a novel human adeno-associated virus capsid with neurotropic properties

Hung-Lun Hsu; Alexander Brown; Anna B. Loveland; Anoushka Lotun; Meiyu Xu; Li Luo; Guangchao Xu; Jia Li; Lingzhi Ren; Qin Su; Dominic J. Gessler; Yuquan Wei; Phillip W. L. Tai; Andrei A. Korostelev; Guangping Gao

doi:10.1038/s41467-020-17047-1

Nature Communications (Jun 2020)

Structural characterization of a novel human adeno-associated virus capsid with neurotropic properties

Hung-Lun Hsu,
Alexander Brown,
Anna B. Loveland,
Anoushka Lotun,
Meiyu Xu,
Li Luo,
Guangchao Xu,
Jia Li,
Lingzhi Ren,
Qin Su,
Dominic J. Gessler,
Yuquan Wei,
Phillip W. L. Tai,
Andrei A. Korostelev,
Guangping Gao

Affiliations

Hung-Lun Hsu: Horae Gene Therapy Center, University of Massachusetts Medical School
Alexander Brown: Horae Gene Therapy Center, University of Massachusetts Medical School
Anna B. Loveland: RNA Therapeutics Institute, University of Massachusetts Medical School
Anoushka Lotun: Horae Gene Therapy Center, University of Massachusetts Medical School
Meiyu Xu: Horae Gene Therapy Center, University of Massachusetts Medical School
Li Luo: Horae Gene Therapy Center, University of Massachusetts Medical School
Guangchao Xu: Horae Gene Therapy Center, University of Massachusetts Medical School
Jia Li: Horae Gene Therapy Center, University of Massachusetts Medical School
Lingzhi Ren: Horae Gene Therapy Center, University of Massachusetts Medical School
Qin Su: Horae Gene Therapy Center, University of Massachusetts Medical School
Dominic J. Gessler: Horae Gene Therapy Center, University of Massachusetts Medical School
Yuquan Wei: State Key Laboratory of Biotherapy, West China Hospital, Sichuan University
Phillip W. L. Tai: Horae Gene Therapy Center, University of Massachusetts Medical School
Andrei A. Korostelev: RNA Therapeutics Institute, University of Massachusetts Medical School
Guangping Gao: Horae Gene Therapy Center, University of Massachusetts Medical School

DOI: https://doi.org/10.1038/s41467-020-17047-1
Journal volume & issue: Vol. 11, no. 1
pp. 1 – 14

Abstract

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Adeno-associated viruses (AAVs) are vehicles for gene therapy in humans, but currently only a limited amount of AAV serotypes is available. Here, the authors identify a novel AAV, AAVv66, and demonstrate enhanced production yields, virion stability, and CNS transduction compared to the clinically approved serotype AAV2.

Published in Nature Communications

ISSN: 2041-1723 (Online)
Publisher: Nature Portfolio
Country of publisher: United Kingdom
LCC subjects: Science
Website: https://www.nature.com/ncomms/

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