New Approach Could Enhance Existing Treatments for Debilitating Genetic Disease
Tuesday, April 6, 2021
The prospect of editing our DNA to treat genetic diseases may have captured the imaginations of scientists and the public in recent years, but that doesn’t mean there aren’t other ways of combating these illnesses. Many promising therapies act not on DNA itself but rather on DNA’s often overlooked cousin, RNA. For instance, experiments in cells performed by IRP researchers have shown promising results or a RNA-targeting therapeutic developed to treat the debilitating genetic disease spinal muscular atrophy.
Discovery Could Improve Therapy for Multiple Autoimmune Diseases
Tuesday, August 20, 2019
Hiding among YouTube’s vast collection of cooking demos and funny cat videos are clips of patients and their advocates designed to raise awareness of specific diseases. It was just such a video that led IRP Senior Investigator Peter Grayson, M.D., M.Sc., to begin studying an extremely rare illness called deficiency of adenosine deaminase 2, or DADA2 for short. The recently published findings of that research could help improve treatment not just for patients with DADA2 but also many more individuals with similar ailments.
Monday, October 24, 2016
This year, members of the National Academy of Medicine elected four NIH Intramural researchers to their ranks, one of the highest honors in science. Learn a bit about each of their research and follow the links to their IRP profiles for more information.