Silencing Huntington's Disease
What is Huntington's disease?
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.
Applications of a therapeutic approach called “gene silencing”, also known as RNAi, have been limited by a lack of simple and efficient methods to deliver these molecules to the brain. Advances in Dr. Neil Aronin, Dr. Phillip Zamore, and Dr. Anastasia Khorova’s labs are making it possible for neurons to internalize chemically modified siRNAs via self-delivery without the need for a carrier. They have identified functional gene silencers (siRNAs) targeting the huntingtin mRNA to specifically silence this diseased gene (mutation of the huntingtin gene is responsible for Huntington's disease).
Image: Julia Alterman, Dr. Anastasia Khvorova's lab. This image shows direct targeting of siRNA to silence the Huntington’s disease gene, in the brain of a mouse after injection of these siRNA molecules into the cerebrospinal fluid.