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Cited by 199Virus-based gene therapy researchRNA Interference and Gene DeliveryViral Infectious Diseases and Gene Expression in Insects

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Adeno-Associated Virus Vectorology, Manufacturing, and Clinical Applications

Grieger, Joshua C., Samulski, R. Jude·2012·Methods in Enzymology
DOI 10.1016/b978-0-12-386509-0.00012-0
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Abstract

Adeno-associated virus (AAV) has emerged as an attractive vector for gene therapy. The benefits of using AAV for gene therapy include long-term gene expression, the inability to autonomously replicate without a helper virus, transduction of dividing and nondividing cells, and the lack of pathogenicity from wild-type infections. A number of Phase I and Phase II clinical trials utilizing AAV have been carried out worldwide (Aucoin et al., 2008; Mueller and Flotte, 2008). A number of challenges have been identified based upon data generated from these clinical trials. These challenges include (1) large scale manufacturing technologies in accordance with current Good Manufacturing Practices (cGMP), (2) tissue specific tropism of AAV vectors, (3) high-quality/high potency recombinant AAV vectors (rAAV), and (4) immune response to AAV capsids and transgene. In this chapter, we will provide an overview of AAV biology, AAV vectorology, rAAV manufacturing, and the current status on the latest rAAV clinical trials.

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Metadata

Authors
Grieger, J. C., & Samulski, R. J.
Affiliations
University of North Carolina at Chapel Hill (US)
ORCID
Samulski, R. Jude
Venue
Methods in Enzymology
Publisher
Elsevier
Published
2012
Volume
507
Pages
229-254
ISSN
0076-6879 (ISSN-L)
DOI
10.1016/b978-0-12-386509-0.00012-0
OpenAlex
W55680868
Cited by
199
References
23
Citations by year
2026: 6 · 2025: 8 · 2024: 9 · 2023: 9 · 2022: 12 · 2021: 13 · 2020: 8 · 2019: 12 · 2018: 12 · 2017: 13 · 2016: 14 · 2015: 26 · 2014: 17 · 2013: 32 · 2012: 7
FWCI
65.29
Type
book chapter
Language
en
Open access
closed
Locations
  • Methods in enzymology on CD-ROM/Methods in enzymology (publishedVersion)
  • PubMed (publishedVersion)
Funders
  • Celladon Corporation
  • National Institutes of Health (5u54ar056953-05)
  • National Institute of Arthritis and Musculoskeletal and Skin Diseases (1U54AR056953)
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
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Viral Infectious Diseases and Gene Expression in Insects
Keywords
adeno-associated virus, recombinant AAV vectors, gene therapy vectors, current good manufacturing practice, AAV tropism, long-term gene expression
MeSH terms
Dependovirus, Hemophilia B, Clinical Trials as Topic, Genetic Vectors, Humans, Technology, Pharmaceutical, Genetic Therapy, Gene Library, Genome, Viral, Cell Culture Techniques, Leber Congenital Amaurosis

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