Neurofilament Regulation

About

Overview Amyotrophic lateral sclerosis (ALS) is a devastating disease with no effective treatment. In patients with ALS, the neurons in their brains degenerate, leading to cognitive and physical breakdown. Other neurodegenerative afflictions include Alzheimer’s disease, Parkinson’s disease and Huntington’s disease. All these diseases share a common feature – structural proteins called neurofilaments (which provide support to neurons) clump up or tangle. The common view is that these tangles are the result of the disease, not the cause. That view is now being challenged. The Invention UW–Madison researchers have demonstrated that neurofilament tangles lead to subsequent degeneration and death of motor neurons in ALS patients. They also discovered that these tangles are caused by the reduced expression of a type of neurofilament mRNA. Thus, neurofilament regulation appears to be a promising target for drug screening and gene therapy. The researchers conducted their studies using motor neurons derived from ALS patients.   Applications Research models, drugs and gene therapies targeting neurofilament regulation   Key Benefits New avenue for treating ALS and potentially other neurodegenerative diseases May lead to earlier diagnosis