Clinical trial recap

So after the initial chaotic excitement, let us try to recap:
two separate gene therapy trials for RPE65 LCA proved the treatment is safe and can work. One was carried out in England, at Moorfields Eye Hospital and the University College London, the other at the Children’s Hospital of Philadelphia in collaboration with The University of Pennsylvania, the Seconda Università of Naples and the Telethon Institute of Genetics and Medicine, also of Naples - go Italy!
Both trials enrolled three patients. The treatment proved safe for all six patients, and it improved vision in all three subjects in the Italian/US trial and in 1 of the London trial.

Both papers were published in the New England Journal of Medicine on the same day. Here are the links to the articles’ abstracts

London trial

US/Italian trial


Certainly a lot to absorb, but the message is simple: the road to future trials for Leber's Congenital Amaurosis and other retinal genetic disorders is now open!