Patients are being enrolled in the first study to test the gene-editing technique known as CRISPR inside the body to try to cure an inherited form of blindness, Newser.com reports.
People with Leber congenital amaurosis have normal eyes but lack a gene that converts light into signals to the brain that enable sight.
The experimental treatment will supply kids and adults with a healthy version of the gene they lack, using a tool that cuts or “edits” DNA in a specific spot, as a one-time treatment that permanently alters the person’s DNA.
Leber congenital amaurosis is the most common cause of inherited childhood blindness, and occurs in 2 to 3 out of every 100,000 births. People with LCA often see only bright light and blurry shapes and eventually can lose all sight.
Parents carry the flawed gene and it can go undetected for generations, then suddenly emerge when an unlucky combination gives a child two copies of it.
Unlike discredited treatments in China on human embryos, this procedure will not produce results that can be inherited.