EYS on" gene editing for retinitis pigmentosa 25

Overview

Project Summary

Inherited Retinal Dystrophies (IRDs) encompass genetic disorders leading to progressive vision loss. Mutations in the Eyes shut homolog (EYS) gene contribute to 5-10% of autosomal recessive IRD cases. Despite the promise of gene therapy, effective treatments for EYS-related IRDs are currently absent. Our research targets a prevalent EYS gene mutation associated with retinitis pigmentosa, a condition devoid of therapeutic options. Using prime editing - a precise molecular tool - we aim to correct genetic errors in patient-derived cells. This initiative addresses the challenge of treating EYS-related IRDs, aiming to fill the current therapeutic gap.

Main Goals

Inherited Retinal Dystrophies (IRDs) encompass genetic disorders leading to progressive vision loss. Mutations in the Eyes shut homolog (EYS) gene contribute to 5-10% of autosomal recessive IRD cases. Despite the promise of gene therapy, effective treatments for EYS-related IRDs are currently absent. Our research targets a prevalent EYS gene mutation associated with retinitis pigmentosa, a condition devoid of therapeutic options. Using prime editing - a precise molecular tool - we aim to correct genetic errors in patient-derived cells. This initiative addresses the challenge of treating EYS-related IRDs, aiming to fill the current therapeutic gap.

Project Details