Gene knockdown in Wet AMD

2025-2027

This project is working to control choroidal neovascularisation in Wet Age-related Macular Degeneration (AMD), using CRISPR Cas9 delivered with adeno-associated vectors (AAVs).

By targeting the VEGF gene to knock down the production of vascular endothelial growth factor, we hope to address the cause of wet AMD, rather than treat the symptoms.  The challenge is that the payload that needs to be delivered to the eye cells is too large for a single AAV: the MacLaren lab is working to adapt a smaller CasX nuclease for this purpose.