Patients are about to be enrolled in the first study to test a gene-editing technique known as CRISPR inside the body to try to cure an inherited form of blindness.
People with the disease have normal eyes but lack a gene that converts light into signals to the brain that enable sight.
Two companies, Editas Medicine and Allergan, will test this in up to 18 people around the United States, including Massachusetts Eye and Ear in Boston, starting this fall. Only one other company, Sangamo Therapeutics, has tried gene editing inside the body, to treat metabolic diseases using a tool called zinc fingers.
The blindness study is for people with one form of Leber congenital amaurosis. It's the most common cause of inherited childhood blindness, occurring in about 2 to 3 of every 100,000 births. People often see only bright light and blurry shapes and eventually can lose all sight.
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