Skip to content

Follow up studies on gene therapy for LCA remain positive despite progression of ONL thinning.

Clinical research from the Scheie Eye Institute, University of Pennsylvania has reported a progression in retinal degeneration despite a clear visual improvement in LCA patients administered with RPE-65 gene augmentation therapy. The findings, published in PNAS (doi/10.1073/pnas.1218933110), suggest that while the gene therapy approach is both efficacious and safe, photoreceptor cells continue to degenerate along a similar time-course to that of untreated patients. The authors of the study conclude that future management of the gene therapy approach may benefit from administering supplemental medication such as neuro-protective agents, anti-apoptotic factors or antioxidants.