CRISPR Gene Editing Will Be Used Inside Humans For the First Time in Treatment for Blindness
Leber congenital amaurosis is a rare inherited eye disease that appears at birth or in the first few months of life and is one of the leading causes of childhood blindness, occurring in about 3 out of 100,000 newborns. Using CRISPR, doctors and researches will inject the gene directly into the light-sensitive cells in an attempt to cure their condition. The trial will consist of 18 patients ranging from children 3 years of age to adults.