A Novel Method for Treating Wet AMD Reversibly with Single Intraocular Injection

Project Summary

The goal of the proposed project is to establish a novel CRISPR-based inducible system for VEGF repression for wet AMD. A CRISPRi system will be used to target VEGF promoter, which can repress VEGF expression without actually editing the gene, therefore avoiding the potential risk of permanent gene editing. Both experiments, cell culture and animal models will be performed to set up the system and test its safety and efficacy. Once the project is complete, we could have a VEGF repression system that combines potency, reversibility and safety, and can be used to treat AMD with just one ocular injection.