NIH SciBites From Home: Developing Gene Therapies for Genetic Diseases

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.

To learn more about the research being done in Shachar's lab, visit

Interested in finding out for yourself what it's like to do research at NIH? Check out our training opportunities to learn about the IRP's programs for students, recent graduates, and postdoctoral fellows:

The SciBites video shorts are produced by [email protected] IRP (Intramural Research Program at the National Institutes of Health). Check out their YouTube channel and visit their website at to learn more about the NIH IRP.
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