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

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

Review article | 更新时间：2024-02-04

- Recombinant adeno-associated virus 8 vector in gene therapy: Opportunities and challenges
- Genes & Diseases Vol. 1, Issue 1, Pages: 283-293(2024)
- 作者机构：
  
  1. Anhui University of Traditional Chinese Medicine,Anhui,Hefei,China,230000
  2. Yangtze Delta Drug Advanced Research Institute, Yangtze Delta Pharmaceutical College,Jiangsu,Nantong,China,226133
  3. InnoStar Bio-tech Nantong Co., Ltd.,Jiangsu,Nantong,China,226133
  4. Shanghai Innostar Bio-Technology Co., Ltd, China State Institute of Pharmaceutical Industry,Shanghai,China,201203
  5. Center for Drug Evaluation, National Medical Products Administration,Beijing,China,100022
- 作者简介：
- 基金信息：
- DOI：10.1016/j.gendis.2023.02.010
  CLC：
- Published：2024，
  
  Published Online：1 January 2024，
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Liyuan Zhao, Zixuan Yang, Minhui Zheng, et al. Recombinant adeno-associated virus 8 vector in gene therapy: Opportunities and challenges. [J]. Genes & Diseases 1(1):283-293(2024)
DOI：

Liyuan Zhao, Zixuan Yang, Minhui Zheng, et al. Recombinant adeno-associated virus 8 vector in gene therapy: Opportunities and challenges. [J]. Genes & Diseases 1(1):283-293(2024) DOI： 10.1016/j.gendis.2023.02.010.

摘要

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.

关键词

Keywords

AAV8Adeno-associated virusGene therapyPrimatesRecombinantRodents

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