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, Alicia A. Brunet, Annie L. Miller, Yashvi Bhatt, Rebekah James, Denise Anderson, Valentina Voigt, João A. Paulo, Mehdi Mirzaei, Melissa M. Mangala, Emilie O Wong, Robyn V. Jamieson, Anai Gonzalez-Cordero, , 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.
bioRxiv · Dec 11, 2025 · PreprintNeuroscience