Huntington's disease is a genetic disease that causes loss of cognition, abnormal movement, and depression. Patients with Huntington's disease generally begin to show symptoms between age 30 and 40, but can exhibit clinical problems in their teenage years. Huntington's disease causes early death, often after many years in high intensity and expensive nursing care. Currently, there is no treatment that alters the course of the disease. My laboratory studies how the mutant gene for Huntington's disease (the gene is called huntingtin) kills certain brain cells called neurons. With my collaborator, Phillip D. Zamore, Ph.D., my laboratory is exploring a promising new therapeutic approach called "gene silencing" or RNAi. The goal of gene silencing is to shut off the mutant huntingtingene (the one that causes the disease), while preserving the normal huntingtin gene. Our initial experiments indicate that our therapeutic strategy is effective and safe in cultured cells and mice. We are currently examining how the gene silencing can be best delivered to people. We study mouse models of Huntington's disease and intend to expand our research to study animals with large brains, as part of the pre-clinical investigations required to develop drugs used to treat people.
For more information, please visit Dr. Neil Aronin Faculty Profile.