The University of California, Los Angeles (UCLA) is conducting a research project under a $345,114 Project Grant award from the National Institute of Neurological Disorders and Stroke (NINDS), funded through the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) program. This 2-year project focuses on developing a high-throughput small molecule screening approach to identify compounds that can lower the endogenous levels of the MSH3 protein, a potential therapeutic target for Huntington's disease and other neurodegenerative disorders involving repeat expansion mutations. The key objectives are to establish a robust primary screening assay using a newly developed mouse model reporter system, as well as to develop secondary assays to further evaluate and prioritize hit compounds. Through this comprehensive screening process, the researchers aim to identify lead compounds with therapeutic potential for Huntington's disease and related neurological conditions.
Generated 3/4/25, 8:32 AM