Developing Gene Therapies for Genetic Diseases

Wednesday, February 24, 2021

For thousands of years, humans have been deliberately altering the environment, their bodies, and the evolution of other organisms. Perhaps the only thing we have been unable to change is our own DNA, but recent breakthroughs in the field of gene therapy have brought even that into the realm of possibility. Shachar Abudi, a graduate student in the Intramural Research Program at the National Institutes of Health, is working on two different ways to use gene therapy to treat a rare genetic disease called Hermansky-Pudlak syndrome. One day, research like hers may enable doctors to help patients with a wide variety of previously untreatable genetic illnesses.