Gene therapy rescue of visual function following onset of disease in retinitis pigmentosa

Researchers based at Columbia University Medical Center, New York have published research findings suggesting that therapeutic rescue in models of recessive retinitis pigmentosa (RP) is possible even after disease onset. The research set out to determine if administration of gene therapy approaches could impact on the course of retinal degeneration in the context of a diseased retina. Given that clinical application of gene therapy in humans will need to address the reality that most RP patients have undergone a degree of rod degeneration when first presenting for medical care, the study aimed to demonstrate that therapy was feasible even when administered after significant disease progression.