Mutations in single genes cause more than 5000 human diseases.
Until recently, doctors have only been able to treat the symptoms of genetic disease, and patients often need to take non-curative drugs for their entire lives. Scientists have known the cause of many genetic disorders for years, but until now there has been no way to cure them. The IGI aims to use the ‘molecular scalpel’ Cas9 to permanently correct disease-causing mutations in cells, animal models, and eventually human patients. Our first major goals are therapies to treat sickle cell disease and a rare familial autoimmune disorder.