Generation of novel AAV serotypes with enhanced infectivity, specificity, and lower toxicity via AAV capsid engineering platform
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.
Authors
- Rui Duan (ORCID: https://orcid.org/0000-0002-5592-5129)
- Ying Fan
- Po‐E Li (ORCID: https://orcid.org/0000-0003-4290-1993)
- Min Guan (ORCID: https://orcid.org/0000-0003-3851-8553)
- In‐Seok Song (ORCID: https://orcid.org/0000-0002-0763-8838)
- Ye Bu
- Junlin Chen
- Zhiyong Dai (ORCID: https://orcid.org/0009-0001-8766-7662)
- Pan Yue (ORCID: https://orcid.org/0000-0002-1336-3324)
- Yujian Zhong (ORCID: https://orcid.org/0000-0002-7043-642X)
- Li Jun He
- Huan Chen (ORCID: https://orcid.org/0000-0001-8648-1631)
- Keqin Tan
- Xin Swanson
Institutions
- South China Normal University (CN)
- Chinese Academy of Sciences (CN)
- 490 BioTech (United States) (US)
- Shenzhen Institutes of Advanced Technology (CN)
Publication Details
- Journal
- Cell and Gene Therapy Insights
- Published
- 2024-12-03
- DOI
- https://doi.org/10.18609/cgti.2024.162
- Primary Topic
- Virus-based gene therapy research
- Type
- article
- Field-Weighted Citation Impact
- 0.00