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Cited by 761Virus-based gene therapy researchRNA Interference and Gene DeliveryNeurogenetic and Muscular Disorders Research

Engineering adeno-associated viruses for clinical gene therapy

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Kotterman, Melissa A., Schaffer, David V.·2014·Nature Reviews Genetics
DOI 10.1038/nrg3742PMC PMC4393649
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Abstract

Clinical gene therapy has been increasingly successful owing both to an enhanced molecular understanding of human disease and to progressively improving gene delivery technologies. Among these technologies, delivery vectors based on adeno-associated viruses (AAVs) have emerged as safe and effective and, in one recent case, have led to regulatory approval. Although shortcomings in viral vector properties will render extension of such successes to many other human diseases challenging, new approaches to engineer and improve AAV vectors and their genetic cargo are increasingly helping to overcome these barriers.

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  • AAV has a shortcoming of limited genetic payload capacity.

    “However, despite its safety and efficacy as a gene therapy vector, AAV has its shortcomings, one being the limited capacity of its genetic payload (Kotterman and Schaffer, 2014).”

    From Evaluating Efficiencies of Dual AAV Approaches for Retinal Targeting (2017) · cites this paper as Kotterman and Schaffer, 2014

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Cited by

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Authors
Kotterman, M. A., & Schaffer, D. V.
Affiliations
University of California, Berkeley (US)
Corresponding author
Schaffer, David V.
ORCID
Venue
Nature Reviews Genetics
Publisher
Springer Science and Business Media LLC
Published
2014-05-20
Volume
15
Issue
7
Pages
445-451
ISSN
  • 1471-0056 (ISSN-L)
  • 1471-0064
DOI
10.1038/nrg3742
PMC
PMC4393649
OpenAlex
W2133171074
Cited by
761
References
19
Citations by year
2026: 29 · 2025: 28 · 2024: 56 · 2023: 51 · 2022: 56 · 2021: 69 · 2020: 60 · 2019: 82 · 2018: 79 · 2017: 96 · 2016: 77 · 2015: 71 · 2014: 6
FWCI
11.03
Type
journal article
Language
en
Open access
Licence
www.springer.com/tdm (tdm)
Locations
  • Nature Reviews Genetics (publishedVersion)
  • PubMed (publishedVersion)
  • Europe PMC (PubMed Central) (acceptedVersion)
  • PubMed Central (acceptedVersion)
  • eScholarship (California Digital Library) (submittedVersion)
Funders
  • National Institutes of Health
  • National Eye Institute (R01EY022975)
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 › Neurogenetic and Muscular Disorders Research
Keywords
adeno-associated virus, adeno-associated virus vectors, gene therapy vectors, vector engineering, rational design, directed evolution, gene delivery, clinical gene therapy
MeSH terms
Dependovirus, Capsid, Clinical Trials as Topic, DNA, Viral, Genetic Engineering, Genetic Vectors, Hemophilia A, Humans, Metabolic Diseases, Muscular Dystrophies, Viral Proteins, Genetic Therapy, Genome, Viral, Directed Molecular Evolution

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