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

Autor:	Zhao L; Anhui University of Traditional Chinese Medicine, Hefei, Anhui 230000, China.; Yangtze Delta Drug Advanced Research Institute, Yangtze Delta Pharmaceutical College, Nantong, Jiangsu 226133, China.; InnoStar Bio-tech Nantong Co., Ltd., Nantong, Jiangsu 226133, China., Yang Z; Shanghai Innostar Bio-Technology Co., Ltd, China State Institute of Pharmaceutical Industry, Shanghai 201203, China., Zheng M; Shanghai Innostar Bio-Technology Co., Ltd, China State Institute of Pharmaceutical Industry, Shanghai 201203, China., Shi L; Shanghai Innostar Bio-Technology Co., Ltd, China State Institute of Pharmaceutical Industry, Shanghai 201203, China., Gu M; Shanghai Innostar Bio-Technology Co., Ltd, China State Institute of Pharmaceutical Industry, Shanghai 201203, China., Liu G; InnoStar Bio-tech Nantong Co., Ltd., Nantong, Jiangsu 226133, China., Miao F; InnoStar Bio-tech Nantong Co., Ltd., Nantong, Jiangsu 226133, China., Chang Y; Shanghai Innostar Bio-Technology Co., Ltd, China State Institute of Pharmaceutical Industry, Shanghai 201203, China., Huang F; Center for Drug Evaluation, National Medical Products Administration, Beijing 100022, China., Tang N; 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.
Jazyk:	angličtina
Zdroj:	Genes & diseases [Genes Dis] 2023 Mar 24; Vol. 11 (1), pp. 283-293. Date of Electronic Publication: 2023 Mar 24 (Print Publication: 2024).
DOI:	10.1016/j.gendis.2023.02.010
Abstrakt:	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. Competing Interests: The authors declare no conflict of interests. (© 2023 The Authors. Publishing services by Elsevier B.V. on behalf of KeAi Communications Co., Ltd.)
Databáze:	MEDLINE
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