Robin Ali, a professor of human molecular genetics at University College London, is leading the world's first human trial of a gene therapy for an eye condition. A total of 12 patients with Leber's Congenital Amaurocis are being treated in the trial. Their photoreceptor cells misfunction due to a faulty RPE65 gene. That is an extremely rare condition, but investigators say that if this trial works they'll be able to use a similar approach to treating about 100 single-gene sight disorders involving far larger numbers of patients. There are now a number of gene therapy studies which are moving from the lab into the clinic. That's a critical milestone for a field that has been regularly plagued by safety concerns. Now Ali and other researchers say they're on their way to putting those old fears into the past as they look forward to new therapies that offer cures.
- here's the report on the project from Time