LOS ANGELES, March 29 (Xinhua) -- U.S. researchers have identified a compound already approved by the U.S. Food and Drug Administration (FDA) that shows promise in lab models for blinding childhood disease, according to a release of the U.S. National Institutes of Health (NIH) on Wednesday.
The NIH research team found the compound could keep light-sensitive photoreceptors alive in three models of Leber congenital amaurosis type 10 (LCA10), an inherited retinal ciliopathy disease that often results in severe visual impairment or blindness in early childhood.
Using a mouse model of LCA10 and two types of lab-created tissues from stem cells known as organoids, the team screened more than 6,000 FDA-approved compounds to identify ones that promoted survival of photoreceptors, the types of cells that die in LCA, leading to vision loss.
The screening identified five potential drug candidates, including Reserpine, an old medication previously used to treat high blood pressure.
Observation of the LCA models treated with Reserpine shed light on the underlying biology of retinal ciliopathies, suggesting new targets for future exploration, said the NIH.
