Is subretinal AAV gene replacement still the only viable treatment option for choroideremia?
Ruofan Connie HanLewis E FryAriel KantorMichelle E McClementsKanmin XueRobert E MacLarenPublished in: Expert opinion on orphan drugs (2021)
The authors conclude that AAV-mediated gene augmentation remains the most effective approach for choroideremia. Given the heterogeneity of CHM mutations and potential risks and benefits, genome-editing approaches currently do not offer significant advantages. Nonsense suppression strategies and antisense oligonucleotides are exciting novel therapeutic options; however, their clinical viability remains to be determined.