Paula I. Fuller-Carter

Pre-clinical validation of AAV-mediated gene therapy for KCNV2 retinopathy improves visual function and expression in mouse and patient models.

It is demonstrated that AAV-mediated gene replacement therapy delivering a codon-optimised human KCNV2 gene subretinally into Kv8.2 knock-out mice significantly restores retinal function, providing the preclinical proof-of-concept for the safety and therapeutic potential of gene therapy for KCNV2 retinopathy.