New gene therapy restores night vision of people with inherited eye disorder

By Nicoletta Lanese published about 15 hours ago

An experimental gene therapy restored the night vision of two people with leber congenital amaurosis.


Two people with a rare inherited eye disorder have had their night vision restored by an experimental gene therapy, researchers say.

These two individuals are part of an ongoing clinical trial(opens in new tab) testing the safety and effectiveness of the new gene therapy, the research team wrote in a report published in October in the journal iScience(opens in new tab). This and additional trials will need to be completed before the therapy can be approved for widespread use, but these early data hint that the treatment can spur "remarkable gains" in patients' night vision, the scientists wrote.

The trial participants have a genetic disorder called leber congenital amaurosis (LCA), which affects an estimated 3 in 100,000 babies, according to University of Florida Health(opens in new tab), one of the institutes involved in developing the therapy.

The disorder primarily affects the retina, the light-sensitive layers of nerve tissue at the back of the eye, and causes severe visual impairment, night or complete blindness within the first two years of life, often from the time of birth, according to the Genetic and Rare Diseases Information Center(opens in new tab). Different forms of LCA impact different genes involved in vision.

More:
https://www.livescience.com/gene-therapy-leber-congenital-amaurosis