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. Still, these early data hints 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 the University of Florida Health(opens in new tab), one of the institutes involved in developing the therapy.