QSP Programs

Huntington’s Disease

  

Huntington’s Disease (HD) is an inherited neurodegenerative disease affecting 4 to 10 people in every 100,000. Despite identification of the causal gene and multiple animal models the pathology of HD is not well understood, and drug-like molecules that can either reduce mutant HTT protein expression, increase its clearance, or prevent mutant HTT-induced cell death have yet to be successfully studied in clinical trials. In collaboration with Dr. Ivet Bahar in the department of Computational and Systems Biology, and Dr. Robert Friedlander in the department of Neurosurgery we have implemented a QSP program to determine the molecular pathogenesis of HD neuronal toxicity and design therapeutic strategies that address the pleiotropic activity of mutant HTT. In collaboration with NCATS, we have identified several molecules which are being used as the basis for computationally identifying key neuroprotective pathways and networks.