The CRISPR gene-editing tool has been used inside the human body for the first time. In a new clinical trial, scientists at the Oregon Health and Science University (OHSU) are testing the technique as a treatment for a genetic mutation that causes blindness.
Ever since it was developed in 2012, CRISPR has shown promise as a potential new way to treat a range of genetic conditions. The tool allows doctors to snip out problematic sections of DNA, such as those that cause disease, and gives the option to replace them with something beneficial.
The new trial, being conducted at OHSU and sponsored by private companies Allergan plc and Editas Medicine, targets a form of Leber congenital amaurosis (LCA). This rare condition is triggered by a genetic mutation that affects the retina, causing patients to either be born blind or lose their sight within the first few years of life.