Discovery of Small Molecule Melanocortin MC4R Correctors to Treat Early-Onset Obesity in MC4R Deficient Patients
Time: 11:30 am
day: Day Two
Details:
- Loss-of-function mutations in the MC4R gene, resulting in impaired trafficking of MC4R to the plasma membrane, leads to MC4R deficiency (MC4R-d) in the paraventricular nucleus of the hypothalamus
- MC4R-d causes severe hyperphagia and is the leading monogenic cause of obesity
- Small molecule pharmacological correctors that stabilize and chaperone MC4R to the cell-surface to restore their function, represents a targeted therapeutic approach for the treatment of MC4R-d associated obesity
