Gene editing for blindness

Scientists have utilised CRISPR gene editing techniques in a clinical trial to treat Leber congenital amaurosis (LCA), a genetic mutation which causes severe visual impairment.

LCA is the most common cause of inherited childhood blindness, and this trial hopes to use advanced gene editing techniques as a one-time permanent treatment to restore sight.

The level of blindness that LCA causes varies, but the majority of people that suffer from it are legally blind with severe sight loss.

Representing the first human in vivo use of CRISPR gene editing, scientists at Allergan and Editas Medicine programmed a harmless virus to enter the body to find and cut out a problematic mutation in the CEP290 gene. Before this, CRISPR had only been used on cells extracted from the body and then reintroduced.