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

Liyuan Zhao; Zixuan Yang; Minhui Zheng; Lei Shi; Mengyun Gu; Gang Liu; Feng Miao; Yan Chang; Fanghua Huang; Naping Tang

doi:10.1016/j.gendis.2023.02.010

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

Genes Dis. 2023 Mar 24;11(1):283-293. doi: 10.1016/j.gendis.2023.02.010. eCollection 2024 Jan.

Authors

Liyuan Zhao^{1

2

3}, Zixuan Yang⁴, Minhui Zheng⁴, Lei Shi⁴, Mengyun Gu⁴, Gang Liu³, Feng Miao³, Yan Chang⁴, Fanghua Huang⁵, Naping Tang^{2

4}

Affiliations

¹ Anhui University of Traditional Chinese Medicine, Hefei, Anhui 230000, China.
² Yangtze Delta Drug Advanced Research Institute, Yangtze Delta Pharmaceutical College, Nantong, Jiangsu 226133, China.
³ InnoStar Bio-tech Nantong Co., Ltd., Nantong, Jiangsu 226133, China.
⁴ Shanghai Innostar Bio-Technology Co., Ltd, China State Institute of Pharmaceutical Industry, Shanghai 201203, China.
⁵ Center for Drug Evaluation, National Medical Products Administration, Beijing 100022, China.

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.

Keywords: AAV8; Adeno-associated virus; Gene therapy; Primates; Recombinant; Rodents.

Publication types

Review