First U.S. Trial Using CRISPR Within the Body Set to Begin

Researchers will attempt to give patients with Leber congenital amaurosis a healthy version of the gene they lack

THURSDAY, July 25, 2019 (HealthDay News) -- 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 the inherited form of blindness known as Leber congenital amaurosis.

Using a tool that cuts or "edits" DNA in a specific spot, researchers will attempt to give pediatric 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, the Associated Press reported.

Leber congenital amaurosis is the most common cause of inherited childhood blindness, occurring in about two to three 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.

AP News Article

Last Updated: