The future of medicine

I study eyes so naturally any exciting news in the visual field catches my eye. Here is a great editorial providing a lay perspective on two articles published yesterday in the New England Journal of Medicine. In short these scientists used a virus to introduce the correct gene, RPE65 into the retinas of patients who had specific genetic mutations in RPE65 associated with Leber's congenital amaurosis, a form of retinal degeneration. The results are quite preliminary, but the implications are amazing. First, the use and administration of this viral vector is safe. This means that other genes can be introduced in the retina. Second, the treatment actually improved the visual acuity of two of the participating patients. This video demonstrates just how effective the treatment was. Click on patient #3 before injection and after injection to see the effects. The beginning shows the actual injections, so some may want to skip to the behavioral results. Absolutely amazing!