Zhao Liyuan, Yang Zixuan, Zheng Minhui, Shi Lei, Gu Mengyun, Liu Gang, Miao Feng, Chang Yan, Huang Fanghua, Tang Naping
Anhui University of Traditional Chinese Medicine, Hefei, Anhui 230000, China.
Yangtze Delta Drug Advanced Research Institute, Yangtze Delta Pharmaceutical College, Nantong, Jiangsu 226133, China.
Genes Dis. 2023 Mar 24;11(1):283-293. doi: 10.1016/j.gendis.2023.02.010. eCollection 2024 Jan.
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.
近年来,基因治疗领域取得了重大突破。腺相关病毒(AAV)是最有前景的基因治疗载体之一,也是递送目的基因的有力工具。在AAV载体中,AAV血清型8(AAV8)因其能高效、稳定地将基因转染到特定组织而备受关注。目前,重组AAV8已广泛应用于多种疾病的基因治疗研究,包括遗传疾病、癌症、自身免疫性疾病和病毒性疾病。本文综述了将AAV8用作基因治疗载体的应用和挑战,旨在为那些致力于将病毒载体应用于基因治疗的人提供有价值的参考资源。