Genetic diseases impact almost 70–80 million people worldwide. Oftentimes, there are limited treatments that doctors can provide, leaving patients with few interventions to manage symptoms.

Recently, though, gene therapy has completely shifted the potential to care for many diseases. Advances in knowledge of responsible genes and nucleic acid technology have revolutionized the ability to specifically edit regions of the genome to correct mutations.

Today (April 18), the Breakthrough Prize Foundation awarded the Life Sciences prize to two teams of five researchers who pioneered gene therapies for two different types of genetic diseases. Physician scientist Jean Bennett, retinal surgeon Albert Maguire, and physician scientist Katherine High from the University of Pennsylvania developed a treatment to cure retinal blindness that is currently in use in the US, Canada, Australia, and Switzerland. Separately, clinical investigator Swee Lay Thein, now at the National Heart, Lung, and Blood Institute, tracked down the gene responsible for continued production of fetal hemoglobin in beta thalassemia and sickle cell disease and, with the help of physician scientist Stuart Orkin at Harvard University, brought this finding from the bench to the bedside.