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用于高效生产腺相关病毒的腺相关病毒基因组修饰

AAV genome modification for efficient AAV production.

作者信息

Asaad Walaa, Volos Polina, Maksimov Denis, Khavina Elena, Deviatkin Andrei, Mityaeva Olga, Volchkov Pavel

机构信息

Genome Engineering Laboratory, Moscow Institute of Physics and Technology, Dolgoprudniy, Russian Federation.

出版信息

Heliyon. 2023 Apr 1;9(4):e15071. doi: 10.1016/j.heliyon.2023.e15071. eCollection 2023 Apr.

Abstract

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.

摘要

腺相关病毒(AAV)是基因治疗中最有效的载体之一。该载体的实验概况显示了其效率和可接受的安全性,这解释了科学家们越来越多地使用它来研究和治疗多种疾病。这些研究需要使用功能正常、纯净且高滴度的载体颗粒。事实上,目前对AAV结构和基因组的了解有助于提高AAV载体的可扩展生产。在这篇综述中,我们总结了通过修饰AAV基因组或细胞内生物过程来优化AAV可扩展生产的最新研究。

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