Gene therapy for choroideremia

Wednesday, February 12 2014 | 00 h 00 min | News

British surgeons have found a new method to heal patients with choroideremia, a genetic disease that can lead to blindness.

This disease, for which no remedy has existed until now, causes pigmentary deposits and a loss of photoreceptors. Young patients see less and less in low-light conditions, until they become blind by adulthood.

In an attempt to heal six young patients, the team of researchers led by Robert McLaren injected the patients with healthy copies of the CHM gene, which was defective in the patients, directly into the retina. The method saved the patients their vision. Despite having retinal detachment, two patients saw their vision improve considerably with regard to low-light vision, sharpness and colours.

The study findings were published in the scientific journal The Lancet. The researchers would like to use a similar method to heal other types of blindness.

Sources:

http://www.acuite.fr/articles.asp?REF=9322

http://www.thelancet.com/journals/lancet/article/PIIS0140-6736(13)62117-0/fulltext