AAV- based vector improvements unrelated to capsid protein modification

Recombinant adeno-associated virus (rAAV) is the leading platform for delivering genetic constructs . To date, three AAV-based gene therapeutic agents have been approved by the FDA and are used in clinical practice. Despite the distinct advantages of gene therapy development, it is clear that AAV ve...

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Bibliographic Details
Published in:	Frontiers in medicine Vol. 10; p. 1106085
Main Authors:	Shitik, Ekaterina M, Shalik, Igor K, Yudkin, Dmitry V
Format:	Journal Article
Language:	English
Published:	Switzerland Frontiers Media S.A 03-02-2023
Subjects:	a transgene cassette adeno-associated virus gene therapy inverted terminal repeats Medicine Rep proteins a transgene cassette inverted terminal repeats gene therapy adeno-associated virus Rep proteins
Online Access:	Get full text
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Summary:	Recombinant adeno-associated virus (rAAV) is the leading platform for delivering genetic constructs . To date, three AAV-based gene therapeutic agents have been approved by the FDA and are used in clinical practice. Despite the distinct advantages of gene therapy development, it is clear that AAV vectors need to be improved. Enhancements in viral vectors are mainly associated with capsid protein modifications. However, there are other structures that significantly affect the AAV life cycle and transduction. The Rep proteins, in combination with inverted terminal repeats (ITRs), determine viral genome replication, encapsidation, etc. Moreover, transgene cassette expression in recombinant variants is directly related to AAV production and transduction efficiency. This review discusses the ways to improve AAV vectors by modifying ITRs, a transgene cassette, and the Rep proteins.
Bibliography:	ObjectType-Article-2 SourceType-Scholarly Journals-1 ObjectType-Feature-3 content type line 23 ObjectType-Review-1 This article was submitted to Gene and Cell Therapy, a section of the journal Frontiers in Medicine Edited by: Chun-Qing Song, Westlake University, China Reviewed by: Pengpeng Liu, University of Massachusetts Medical School, United States; Arun Srivastava, University of Florida, United States; Chengwen Li, University of North Carolina at Chapel Hill, United States; Yue Zhang, Zhejiang University, China
ISSN:	2296-858X 2296-858X
DOI:	10.3389/fmed.2023.1106085