More Hope than Ever!

Breaking research news – Gene Therapy provides vision to nearly blind young adults!

​Three young adults with virtually no vision can now read several lines on an eye chart and see better in dimly lit settings thanks to an innovative gene therapy aiming to reverse blindness in a severe form of retinitis pigmentosa known as Leber congenital amaurosis or LCA. One person was even able to better navigate an obstacle course several weeks after receiving the therapy.

The three individuals are participating in a Phase I clinical trial at The Children’s Hospital of Philadelphia.
This is an incredible milestone in curing blindness, and this advancement will help pave the way for the development of gene therapies to treat and cure a variety of retinal diseases including Usher syndrome.
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Doctors working on Usher syndrome research are working to extend this incredible breakthrough to Usher syndrome. There is great hope that clinical trials for similar gene therapy for Usher syndrome is right around the corner. Future funding is needed for this important research to continue.