This autumn, 18 blind Americans with Leber's congenital haze type 10 will undergo a clinical trial of a new treatment. EDIT-101 is a gene editing therapy developed by Editas Medicine and Allergan.
This is not the first time that gene editing technology has entered a formal human clinical trial, but because EDIT-101 needs to be injected directly into the retina of patients, it is expected to become the world.The first is CRISPR/Cas9 gene editing therapy that is directly used in humans.Leber congenital amaurosis (LCA) is a serious hereditary retinopathy and one of the main causes of congenital blindness in children. Infants with this disease lose binocular cone cell function rapidly from birth to under one year of age until they are completely blind. Prior to human clinical trials, the technique had been tested in vitro in retinal tissue, in mice and in primate safety.