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Design Strategies of Adeno-Associated Virus (AAV) Vectors in Gene Therapy
  • +4
  • yafeng lv,
  • hao zhang,
  • shuyue li,
  • xiongzhou zhang,
  • hengxin tan,
  • jianlin yang,
  • chunyu cao
yafeng lv
China Three Gorges University

Corresponding Author:[email protected]

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xiongzhou zhang
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hengxin tan
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jianlin yang
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chunyu cao
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Abstract

Adeno-associated viruses (AAV) have attracted significant attention in the field of gene and cell therapy due to highly effective delivery of therapeutic genes into human cells. At present, three AAV-based gene therapy drugs (Glybera, Luxturna and Zolgensma) for the treatment of genetic diseases have been approved by the European Medicines Agency (EMA) and the U.S Food and Drug Administration (FDA) during 2012-2021. In order to make the AAV vector better meet the needs of gene therapy, researchers have carried out a series of optimization and modification on the AAV vectors, which mainly include the modification of the AAV capsid protein, the optimization of the promoter, and Codon optimization for transfer genes. The use of AAV vectors to treat single-gene disorders has become more feasible than ever, but the development of new AAV-based gene therapy drugs still faces many challenges, Such as immunological barriers, large-scale production etc. This article will briefly review the strategies of AAV vectors design and the key challenges in recent years.