Pre-clinical validation of a novel AAV-mediated gene therapy for KCNV2 retinopathy improves visual function in a mouse model and expression in patient organoids
Rabab Rashwan, Paula I. Fuller-Carter, Xin Ru Lim…·Lívia S. Carvalho
This paper demonstrates that AAV-mediated gene therapy restores retinal function and KCNV2 expression in a mouse model of KCNV2 retinopathy and human retinal organoids.