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Review Article | Open Access

Recombinant adeno-associated virus 8 vector in gene therapy: Opportunities and challenges

Liyuan Zhaoa,b,dZixuan YangcMinhui ZhengcLei ShicMengyun GucGang LiudFeng MiaodYan ChangcFanghua Huange( )Naping Tangb,c( )
Anhui University of Traditional Chinese Medicine, Hefei, Anhui 230000, China
Yangtze Delta Drug Advanced Research Institute, Yangtze Delta Pharmaceutical College, Nantong, Jiangsu 226133, China
Shanghai Innostar Bio-Technology Co., Ltd, China State Institute of Pharmaceutical Industry, Shanghai 201203, China
InnoStar Bio-tech Nantong Co., Ltd., Nantong, Jiangsu 226133, China
Center for Drug Evaluation, National Medical Products Administration, Beijing 100022, China

Peer review under responsibility of Chongqing Medical University.

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Abstract

In recent years, significant breakthroughs have been made in the field of gene therapy. Adeno-associated virus (AAV) is one of the most promising gene therapy vectors and a powerful tool for delivering the gene of interest. Among the AAV vectors, AAV serotype 8 (AAV8) has attracted much attention for its efficient and stable gene transfection into specific tissues. Currently, recombinant AAV8 has been widely used in gene therapy research on a variety of diseases, including genetic diseases, cancers, autoimmune diseases, and viral diseases. This paper reviewed the applications and challenges of using AAV8 as a vector for gene therapy, with the aim of providing a valuable resource for those pursuing the application of viral vectors in gene therapy.

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Genes & Diseases
Pages 283-293

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Cite this article:
Zhao L, Yang Z, Zheng M, et al. Recombinant adeno-associated virus 8 vector in gene therapy: Opportunities and challenges. Genes & Diseases, 2024, 11(1): 283-293. https://doi.org/10.1016/j.gendis.2023.02.010

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Received: 20 October 2022
Revised: 26 December 2022
Accepted: 08 February 2023
Published: 24 March 2023
© 2023 The Authors.

This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).