1. A novel AAV Vector for gene therapy of RPE-related retinal degenerative diseases via intravitreal delivery.
- Author
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Gong, Yajun, Huang, Xianyu, Tu, Tianxiang, Chu, Cenfeng, Xian, Chunrui, Yuan, Yushun, Fu, Xin, Li, Ruobi, Zhong, Guisheng, and Zhou, Xiaolai
- Subjects
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MACULAR degeneration , *INTRAVITREAL injections , *TREATMENT effectiveness , *RETINAL degeneration , *RETINAL diseases , *RETINAL ganglion cells - Abstract
The article in the journal "Molecular Neurodegeneration" discusses the development of a novel AAV vector, AAV206, for gene therapy of RPE-related retinal degenerative diseases via intravitreal delivery. The study highlights the high specificity and efficiency of AAV206 in transducing RPE cells, with lower retinal toxicity and immunogenicity compared to conventional AAV2 vectors. Additionally, the research demonstrates that AAV206-sFLT shows superior efficacy in inhibiting CNV formation in a laser-induced CNV mouse model compared to AAV2-sFLT. The findings suggest that AAV206 holds promise as a valuable tool for treating RPE-related retinal degenerative diseases. [Extracted from the article]
- Published
- 2024
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