AAV genome modification for efficient AAV production

The adeno-associated virus (AAV) is one of the most potent vectors in gene therapy. The experimental profile of this vector shows its efficiency and accepted safety, which explains its increased usage by scientists for the research and treatment of a wide range of diseases. These studies require usi...

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Bibliographic Details
Published in:Heliyon 2023-04, Vol.9 (4), p.e15071-e15071, Article e15071
Main Authors: Asaad, Walaa, Volos, Polina, Maksimov, Denis, Khavina, Elena, Deviatkin, Andrei, Mityaeva, Olga, Volchkov, Pavel
Format: Article
Language:English
Subjects:
AAP
AAV
AAV production
ITR
ITRs
MAAP
Review
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Summary:The adeno-associated virus (AAV) is one of the most potent vectors in gene therapy. The experimental profile of this vector shows its efficiency and accepted safety, which explains its increased usage by scientists for the research and treatment of a wide range of diseases. These studies require using functional, pure, and high titers of vector particles. In fact, the current knowledge of AAV structure and genome helps improve the scalable production of AAV vectors. In this review, we summarize the latest studies on the optimization of scalable AAV production through modifying the AAV genome or biological processes inside the cell.
ISSN:2405-8440
2405-8440
DOI:10.1016/j.heliyon.2023.e15071