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

被引：20

作者：

Zhao, Liyuan ^{[1
,2
,4
]}

Yang, Zixuan ^{[3
]}

Zheng, Minhui ^{[3
]}

Shi, Lei ^{[3
]}

Gu, Mengyun ^{[3
]}

Liu, Gang ^{[4
]}

Miao, Feng ^{[4
]}

Chang, Yan ^{[3
]}

Huang, Fanghua ^{[5
,7
]}

Tang, Naping ^{[2
,3
,6
]}

机构：

[1] Anhui Univ Tradit Chinese Med, Hefei 230000, Anhui, Peoples R China

[2] Yangtze Delta Pharmaceut Coll, Yangtze Delta Drug Adv Res Inst, Nantong 226133, Peoples R China

[3] Shanghai Duomirui Biotechnol Ltd, China State Inst Pharmaceut Ind, Shanghai 201203, Peoples R China

[4] InnoStar Biotech Nantong Co Ltd, Nantong 226133, Peoples R China

[5] Natl Med Prod Adm, Ctr Drug Evaluat, Beijing 100022, Peoples R China

[6] Shanghai Innostar Biotech Co Ltd, China State Inst Pharmaceut Ind, 199 Guoshoujing Rd, Shanghai 201203, Peoples R China

[7] Natl Med Prod Adm, Ctr Drug Evaluat, 128 Jianguo Rd, Beijing 100022, Peoples R China

来源：

GENES & DISEASES | 2024年 / 11卷 / 01期

关键词：

AAV8; Adeno-associated virus; Gene therapy; Primates; Recombinant; Rodents; HEMOPHILIA-A MICE; NEUTRALIZING ANTIBODIES; EFFICIENT TRANSDUCTION; LIVER TRANSDUCTION; PERIPHERAL VEIN; RHESUS-MONKEYS; VIRAL VECTORS; MOUSE MODEL; AAV VECTOR; SEROTYPE-8;

D O I：

10.1016/j.gendis.2023.02.010

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

In recent years, significant breakthroughs have been made in the field of gene ther-apy. 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 va-riety 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 ther-apy, with the aim of providing a valuable resource for those pursuing the application of viral vectors in gene therapy. 2023 The Authors. Publishing services by Elsevier B.V. on behalf of KeAi Communications Co., Ltd. This is an open access article under the CC BY-NC-ND license (http://creativecommons. org/licenses/by-nc-nd/4.0/).

引用

页码：283 / 293

页数：11

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