CRISPR to be Used to Fight a Type of Blindness

From the WebMD Archives

July 26, 2019 -- In a U.S. first, a clinical trial to begin this fall will use the inside-the-body gene-editing technique CRISPR to try to cure illness. Doctors hope to use the cutting-edge technique to cure a type of inherited form of blindness called Leber congenital amaurosis.

Even though they have normal eyes, patients with this type of blindness lack a gene that turns light into signals to the brain that enable sight, the Associated Press reported.

Using a tool that cuts or "edits" DNA in a specific spot, researchers will attempt to give child and adult patients a healthy version of the gene they lack. The trial will include 18 people across the United States and will be conducted by two companies, Editas Medicine and Allergan.

Leber congenital amaurosis is the most common cause of inherited childhood blindness, occurring in about 2 to 3 of every 100,000 births.

The only other trial to use gene editing inside the body was to treat metabolic diseases. That was done by a company called Sangamo Therapeutics, the AP reported.