The Charcot-Marie-Tooth Research Foundation has awarded Chris Lorson, PhD, professor in the MU College of Veterinary Medicine Department of Veterinary Pathobiology and associate dean for Research and Graduate Studies, and Michael Garcia, PhD, an associate professor of biological sciences in MU’s College of Arts and Sciences, a $200,000 grant for their research focusing on CMT disease. Their study is “A precision medicine-based approach for CMT2E.”
According to the CMTRF website, CMT is a disease involving the peripheral nerves that branch out from the brain and spinal cord to other parts of the body, including the arms, hands, legs and feet. Lorson and Garcia’s research focuses on treatments for specific types of CMT instead of a one-size-fits-all that would target the average patient.
A press release from CMTRF highlights the process and goals of this research, stating, “Drs. Lorson and Garcia will develop and test a new gene therapy approach that will both silence the abnormal gene and simultaneously replace it with genetic material that will produce normal protein. The research team will test this approach in an animal model of CMT2E that is already well understood, making it suitable for this proof-of-concept project. If the approach is successful, it could potentially be used for other forms of CMT as well. In fact, it is possible this approach could be used for many CMT-causing mutations that require both silencing of a mutated gene and replacement with normal protein, whether the mutation is currently known or has yet to be discovered. The ability to silence and replace genes, regardless of the specific mutation, is what makes this a precision medicine approach.”