Genomics
Institute
Menu
Programs
Human Health
Mutations in single genes cause more than 5000 human diseases.
Doctors can only treat the symptoms of genetic disease and patients often need to take non-curative drugs for their entire life. 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.
46
Principal Investigators
37
Research Projects
33
Published Papers
Key Goals
Meet an IGI Scientist
Press ReleasesCOVID-19
Press ReleasesCOVID-19
Press ReleasesCOVID-19
Press ReleasesCOVID-19