Kennedy’s disease is a neuromuscular disorder primarily affecting men that currently has no effective treatment. It is caused by a mutation in a gene known as Androgen Receptor, resulting in weakening of muscles in the arms and legs and head region. Studies have shown that when expression of the mutant Androgen Receptor is reduced, particularly in skeletal muscle, the disease phenotype is improved in animal models of disease, supporting the idea for reducing Androgen Receptor expression in muscle for the treatment of Kennedy’s Disease.

I have developed an innovative method to reduce expression of the problematic mutant Androgen Receptor gene specifically in muscles to reduce the likelihood of the therapy impacting unaffected organs. My research is now focused on testing this system in normal mice, in mice with Kennedy’s disease and in cells collected from Kennedy’s Disease patients. This is to ensure that there are no unexpected side effects of our therapeutic and to assess the beneficial effects of our therapeutic in mice with Kennedy’s Disease. The results of this project will allow for further understanding and for the development of an innovative therapy for this rare disorder. Furthermore, the progression of these technologies will permit further investigation of potential muscle-targeted therapeutics for other neuromuscular diseases in addition to Kennedy’s Disease.