A genetic treatment for blindness could soon be on the way
A novel treatment for Leber congenital amaurosis has shown to be effective in improving vision in affected patients.
New research presented at the 121st Annual Meeting of the American Academy of Ophthalmology (AAO) (11–14 November 2017; LA, USA), has demonstrated that a novel gene therapy is effective in partially restoring vision in individuals affected by Leber congenital amaurosis (LCA).
LCA is a rare disease that affects approximately 1 in 80,000 individuals and can be caused by one or of 19 genes. The disease begins in infancy, progresses slowly and eventually leads to blindness. There are currently no effective treatments for the disease.
The gene therapy presented at the meeting is termed voretigene neparvovec. The therapy uses a genetically-modified virus to carry healthy versions of the gene into the retina, which then helps ameliorate the symptoms of LCA. The therapy is currently under review by the FDA, with an advisory committee to the FDA unanimously endorsing the treatment. The FDA isn't obligated to follow the recommendations of its advisory committees and it will make its final decision before January 2018.
Approval could open the door for other gene therapies that could eventually treat the more than 225 genetic mutations known to cause blindness. It could also be applied to retinitis pigmentosa, another inherited retinal disease caused by a defective gene. Alternatively in the future, gene therapy could possibly provide key proteins needed to restore vision in more common diseases such as age-related macular degeneration.