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Adeno-associated virus vector as a platform for gene therapy delivery
Open accessAbstract
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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In the citing paper
“A primary immune response to the treatment may be triggered by the presence of pre-existing neutralizing antibodies (NABs) against rAAV capsids, due to previous naturally-occurring exposure to AAV [1, 3].”
From Characterization of anti-AAV2 neutralizing antibody levels in sheep prior to and following intravitreal AAV2.7m8 injection (2024) · cites this paper as [3]
Experimental — accuracy not yet measuredIn the citing paper
“However, host immune responses against AAV vectors and delivered transgene impact effective and stable transgene expression [10, 11].”
From Immunogenicity of Recombinant Adeno-Associated Virus (AAV) Vectors for Gene Transfer (2023) · cites this paper as [11]
In the citing paper
“One of the first approaches to show gene transfer in mouse retina used rAAV vectors to deliver a reporter gene by subretinal injection into an adult mouse retina [62].”
From The Ocular Gene Delivery Landscape (2021) · cites this paper as [62]
In the citing paper
“The therapeutic payload must be under 5 kb and must include the regulatory elements, such as the promoter and polyadenylation signal [61,62].”
From The Ocular Gene Delivery Landscape (2021) · cites this paper as [62]
In the citing paper
“The latter pathway results in unsuccessful transduction, and capsid-derived peptides are presented to CTL by MHC class I molecules, provoking elimination of transduced cells and loss of transgene expression (85).”
From Immunomodulation in Administration of RAAV: Preclinical and Clinical Adjuvant Pharmacotherapies (2021) · cites this paper as [85]
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“These features have made rAAVs the most widely used vehicles in the gene therapy fi eld, especially for treating ocular disease [2, 3]. rAAVs have demonstrated ef fi cacy in clinical trials for inherited retinal dystrophies, including Leber ’ s congenital amaurosis (LCA), chor-oideremia, and X-linked…”
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Metadata
- Authors
- Wang, D., Tai, P. W. L., & Gao, G.
- Affiliations
- University of Massachusetts Chan Medical School (US)
- Corresponding author
- Gao, Guangping
- Venue
- Nature Reviews Drug Discovery
- Published
- 2019-02-01
- Volume
- 18
- Issue
- 5
- Pages
- 358-378
- ISSN
- 1474-1776 (ISSN-L)
- 1474-1784
- 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
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
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