The power of CRISPR gene editing provides the exciting potential to cure retinal diseases with no available treatment. Genetic mutations cause Best disease, a retinal disorder that leads to progressive vision loss. We are optimizing the CRISPR-Cas9 genome engineering system to efficiently correct the disease-causing mutations in retinal cells while avoiding unintended genetic changes. We grow retinal cells from iPSCs, allowing us to test editing methods in the patient’s own cells before a clinical trial. We are exploring delivery methods such as transplanting ex vivo edited retinal cells or injecting CRISPR editing molecules directly under a patient’s retina to fix the disease-causing mutation. These proof-of-concept studies will provide the first steps toward potentially vision-restoring therapeutic editing.