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Generation of novel AAV serotypes with enhanced infectivity, specificity, and lower toxicity via AAV capsid engineering platform

Year: 2024

Type: article

Abstract: AAV-based gene therapies pose a number of challenges to developers, including ineffective uptake to target tissues, off-target effects and toxicity, and virus neutralization. Therefore, naturally occurring AAV serotypes cannot fulfill all the requirements of targeted gene therapy. Capsid engineering can provide a solution to this issue by enabling successful development of novel AAV serotypes with enhanced specificity, infectivity, and reduced toxicity. In this article, a capsid engineering platform is described, along with study data on promising AAV variants generated using this approach.

Source: Cell and Gene Therapy Insights

DOI: 10.18609/cgti.2024.162

Authors: Ye Bu, Yue Pan, Yujian Zhong, Huan Chen, Zhiyong Dai, Ying Fan, Junlin Chen, Keqin Tan, Rui Duan, Min Guan, Irene Song, Luyan He, Xin Swanson, Paul Li

Institutions: South China Normal University, Chinese Academy of Sciences, Shenzhen Institutes of Advanced Technology

Language: English

Topic: Virus-based gene therapy research

Subfield: Genetics

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: hybrid

OpenAlex ID: W4417359181

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