Center for Hereditary Retinal Degenerations
Our Center aims to provide better sight for people living with inherited retinal diseases (IRDs) such as retinitis pigmentosa, Stargardt disease, Leber congenital amaurosis, blue cone monochromacy, choroideremia and many others. Co-director Dr. Aleman provides clinical care to IRD patients who carry the burden of progressive vision loss that is currently not treatable. Co-directors Dr. Cideciyan and Dr. Aleman perform world-class research in clinical trials and in the laboratory to make progress towards treatments.
New 2026 Publications
We just published two papers: our evaluation of toxicity and efficacy of BEST1 gene therapy which is currently in human clinical trials, and our latest review of the field of inherited retinal degenerations.
Gray A, Kwok JC, Sato Y, Assenmacher CA, Choudhary M, Jayagopal A, Hua P, Ying GS, Swider M, Sumaroka A, Cideciyan AV, Gustavo AD, Beltran WA. Evaluation of the toxicity and efficacy of an adeno-associated viral vector expressing BEST1 delivered by subretinal injection in a canine model of human bestrophinopathy. Hum Gene Ther. 2026:10430342261458076.
Uyhazi KE, O'Neil EC, Aleman TS, Cideciyan AV. Inherited retinal degenerations: clinical phenotypes and emerging therapies. Lancet. 2026:S0140-6736(26)00766-X.