[Comment] Gene therapy for RPE65-mediated inherited retinal dystrophy completes phase 3

The eye is an attractive organ for gene therapy because of the small amount of tissue that needs to be transfected, surgical accessibility for gene delivery, and the inbuilt control of the second eye. However, despite these benefits, no licensed ocular gene therapy exists for patients with otherwise incurable eye diseases. Stephen Russell and colleagues1 are therefore to be congratulated for doing the first, to our knowledge, phase 3 randomised controlled trial of a gene therapy for a genetic disease.
Source: LANCET - Category: General Medicine Authors: Tags: Comment Source Type: research