The six patients had severe vision loss from choroideremia, a form of retinopathy that is genetic. The disease is fairly rare and manifests only in males. The cells at the back of the eye gradually die, leading to gradual vision loss and ultimately, blindness. Working copies of the faulty gene were injected into retina cells, along with a fluid to halt further degeneration.
After the surgery, the patients found their vision not only stabilized, but also improved. The patients with advanced retinopathy were able to read an optician’s chart significantly better and experienced improved function day-to-day. Patients whose vision loss was not as advanced saw improvements in night vision, which allowed them to see in dimmer light and drive at night. One patient reported being able to see foliage more vividly and stars in the sky for the first time in decades.
If these initial patients continue to improve, this surgery would be ideal for younger choroideremia patients in order to prevent vision loss. The treatment targets the same cells that are damaged by macular degeneration. Therefore, this work may lead to treatments in the future for diseases of the eye, perhaps even glaucoma.
This work was lead by Professor MacLaren at Oxford University in England.