Our lab is dedicated to developing therapeutics for Huntington’s disease. Huntington’s disease (HD) is a devastating neurodegenerative disorder characterized by progressive symptoms which include motor impairments, cognitive decline, and psychiatric symptoms such as depression and anxiety. Huntington’s disease is caused by expansion of the CAG repeat region in Exon 1 of the Huntingtin gene. While most people have approximately 20 CAG repeats, Huntington’s disease patients have more than 35 repeats. Expansion of the CAG repeat region results in production of a mutant protein which is toxic to cells.
Projects in the lab are focused on:
reducing the amount of mutant protein using RNA interference
increasing clearance of the mutant protein by modifying cellular degradation processes
nvestigating downstream pathways that are disrupted during the course of the disease.
We have active collaborations with a variety of researchers focused on delivery and optimization of methods for reducing Huntingtin in the brain.
