This $195,000 federal Project Grant award from the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) will support research to determine the mechanism of action of small molecule splicing modulators for the treatment of Huntington's disease. The University of Colorado-Denver, a major federal grant and contract recipient, will conduct this research using a combination of cryogenic electron microscopy and biochemical approaches. The goal is to better understand how these splicing modulators can reduce levels of the huntingtin protein, which is the root cause of Huntington's disease, in order to facilitate further development of these compounds as potential therapies. This 2-year award, running from April 1, 2025 to March 31, 2027, aims to overcome challenges in the development of splicing modulators for central nervous system diseases.
Generated 3/25/25, 2:57 AM