Cited by 2,444Virus-based gene therapy researchRNA Interference and Gene DeliveryNeurogenetic and Muscular Disorders Research

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Adeno-associated virus vector as a platform for gene therapy delivery

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Wang, Dan, Tai, Phillip W. L., Gao, Guangping·2019·Nature Reviews Drug Discovery

DOI 10.1038/s41573-019-0012-9PMC PMC6927556

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Abstract

Adeno-associated virus (AAV) vectors are the leading platform for gene delivery for the treatment of a variety of human diseases. Recent advances in developing clinically desirable AAV capsids, optimizing genome designs and harnessing revolutionary biotechnologies have contributed substantially to the growth of the gene therapy field. Preclinical and clinical successes in AAV-mediated gene replacement, gene silencing and gene editing have helped AAV gain popularity as the ideal therapeutic vector, with two AAV-based therapeutics gaining regulatory approval in Europe or the United States. Continued study of AAV biology and increased understanding of the associated therapeutic challenges and limitations will build the foundation for future clinical success.

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See all 2,444

Authors: Wang, D., Tai, P. W. L., & Gao, G.
Affiliations: University of Massachusetts Chan Medical School (US)
Corresponding author: Gao, Guangping
ORCID: Wang, Dan
Tai, Phillip W. L.
Gao, Guangping
Venue: Nature Reviews Drug Discovery
Publisher: Springer Science and Business Media LLC
Published: 2019-02-01
Volume: 18
Issue: 5
Pages: 358-378
ISSN: 1474-1776 (ISSN-L)
1474-1784
DOI: 10.1038/s41573-019-0012-9
PMC: PMC6927556
OpenAlex: W2913212173
Cited by: 2,444
References: 319
Citations by year: 2026: 314 · 2025: 431 · 2024: 469 · 2023: 385 · 2022: 342 · 2021: 295 · 2020: 168 · 2019: 39
FWCI: 27.96
Type: journal article
Language: en
Open access: green
www.ncbi.nlm.nih.gov
Licence: www.springer.com/tdm (tdm)
Locations: Nature Reviews Drug Discovery (publishedVersion)
PubMed (publishedVersion)
Europe PMC (PubMed Central) (acceptedVersion)
PubMed Central (acceptedVersion)
Funders: National Heart, Lung, and Blood Institute (P01HL131471, UG3HL147367)
National Institute of Neurological Disorders and Stroke (R01NS076991)
Topics: Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
Health Sciences › Medicine › Genetics (clinical) › Neurogenetic and Muscular Disorders Research
Keywords: adeno-associated virus vectors, gene therapy, gene delivery, AAV capsid, gene replacement, gene silencing, genome editing, clinical gene therapy
MeSH terms: Dependovirus, Animals, Genetic Vectors, Humans, Genetic Therapy, Gene Transfer Techniques

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Adeno-associated virus vector as a platform for gene therapy delivery

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