Cited by 225Virus-based gene therapy researchRNA Interference and Gene DeliveryImmunotherapy and Immune Responses

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Basic Biology of Adeno-Associated Virus (AAV) Vectors Used in Gene Therapy

Balakrishnan, Balaji, Jayandharan, Giridhara·2014·Current Gene Therapy

DOI 10.2174/1566523214666140302193709

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Abstract

Adeno-associated virus (AAV) based vectors have emerged as important tools for gene therapy in humans. The recent successes seen in Phase I/II clinical trials have also highlighted the issues related to the host and vector-related immune response that preclude the universal application of this promising vector system. A fundamental insight into the biological mechanisms by which AAV infects the host cell and a thorough understanding of the immediate and long-lived cellular responses to AAV infection is likely to offer clues and help design better intervention strategies to improve the therapeutic efficiency of AAV vectors. This article reviews the biology of AAV-host cellular interactions and outlines their application in the development of novel and improved AAV vector systems.

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“Based on in vivo studies, vectors specific to retina (AAV2, AAV4 and AAV5) [64-68], brain (AAV2, AAV5, AAV8, AAV9 and AAVrh10) [69-78], cardiac tissue (AAV1, AAV2, AAV6, AAV8 and AAV9) [79-83], liver (AAV1, AAV2, AAV5, AAV6, AAV7, AAV8, AAV9 and AAV10) [49, 84-93] and lungs (AAV1, AAV2, AAV5 and AAV9) [86-89, 92-98] h…”
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“Based on in vivo studies, vectors specific to retina (AAV2, AAV4 and AAV5) [64-68], brain (AAV2, AAV5, AAV8, AAV9 and AAVrh10) [69-78], cardiac tissue (AAV1, AAV2, AAV6, AAV8 and AAV9) [79-83], liver (AAV1, AAV2, AAV5, AAV6, AAV7, AAV8, AAV9 and AAV10) [49, 84-93] and lungs (AAV1, AAV2, AAV5 and AAV9) [86-89, 92-98] h…”
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Authors: Balakrishnan, B., & Jayandharan, G.
Affiliations: Bentham Science Publishers (United Arab Emirates) (AE)
Christian Medical College, Vellore (IN)
ORCID: Balakrishnan, Balaji
Jayandharan, Giridhara
Venue: Current Gene Therapy
Publisher: Bentham Science Publishers Ltd.
Published: 2014-05-31
Volume: 14
Issue: 2
Pages: 86-100
ISSN: 1566-5232 (ISSN-L)
DOI: 10.2174/1566523214666140302193709
OpenAlex: W2142695353
Cited by: 225
References: 186
Citations by year: 2026: 6 · 2025: 29 · 2024: 22 · 2023: 24 · 2022: 24 · 2021: 28 · 2020: 37 · 2019: 18 · 2018: 13 · 2017: 13 · 2016: 7 · 2015: 1 · 2014: 3
FWCI: 5.24
Type: journal article
Language: en
Open access: closed
Locations: Current Gene Therapy (publishedVersion)
PubMed (publishedVersion)
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 › Immunology and Microbiology › Immunology › Immunotherapy and Immune Responses
Keywords: adeno-associated virus, adeno-associated virus vectors, gene therapy vectors, host immune response, vector immunogenicity, vector design
MeSH terms: Dependovirus, Genetic Vectors, Humans, Genetic Therapy, Clinical Trials, Phase I as Topic, Clinical Trials, Phase II as Topic, Gene Transfer Techniques, Host-Pathogen Interactions

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Basic Biology of Adeno-Associated Virus (AAV) Vectors Used in Gene Therapy

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