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Review Article

Genetic Engineering of AAV Capsid Gene for Gene Therapy Application

Author(s): Yunbo Liu, Xu Zhang and Lin Yang*

Volume 20, Issue 5, 2020

Page: [321 - 332] Pages: 12

DOI: 10.2174/1566523220666200930105521

Price: $65

Abstract

Adeno-associated virus (AAV) is a promising vector for in vivo gene therapy because of its excellent safety profile and ability to mediate stable gene expression in human subjects. However, there are still numerous challenges that need to be resolved before this gene delivery vehicle is used in clinical applications, such as the inability of AAV to effectively target specific tissues, preexisting neutralizing antibodies in human populations, and a limited AAV packaging capacity. Over the past two decades, much genetic modification work has been performed with the AAV capsid gene, resulting in a large number of variants with modified characteristics, rendering AAV a versatile vector for more efficient gene therapy applications for different genetic diseases.

Keywords: Adeno-associated virus, capsid gene, genetic modification, tissue tropism, neutralizing antibody, DNA.

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