In this new CMT Research Foundation-funded project, Drs. Lorson and Garcia at the University of Missouri 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.
CMT Research Foundation Launches New Research Project to Design Precision Medicine Approach for Charcot-Marie-Tooth Disease
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