X-linked RP caused by RPGR mutations, an attractive target for gene therapy because of its clinical severity and large number of patients.

By: Glenn Yiu, MD, PhD - Department of Ophthalmology & Vision Science, University of California, Davis, Sacramento, California Ophthalmology

Shows promise in stem cell model of retinitis pigmentosa Although the 2017 FDA approval of Spark Therapeutics’ gene therapy Luxturna was...