CMT Research Foundation invests in research projects to develop treatments for CMTs
The CMT Research Foundation (CMTRF) has invested in a research project based at Nationwide Children’s Hospital, US, to develop new vehicles for delivering gene therapies to treat several types of Charcot-Marie-Tooth disease (CMT). The goal of the new initiative is to increase the safety and effectiveness of gene therapies for treating CMTs, especially CMT1A, the most common kind that affects 50% of individuals with diseases that damage nerves.
CMT is a set of genetic disorders that damage the peripheral nerves, which are located outside the main central nervous system. It is recognized as a rare peripheral neuropathy disease that affects around three million individuals worldwide. To develop safe and efficient gene therapies for CMT, a therapeutic genetic payload that specifically targets Schwann cells a subset of glial cells that aid in the formation of the myelin sheath around nerve fibers must be delivered into the peripheral nervous system. This is necessary for CMT types CMT1A, 1B, 4C, 4J, and 1X. Currently, gene treatments are carried out utilizing adeno-associated viral (AAV) vectors. When creating AAV-based gene therapy for CMT, Afrooz Rashnonejad, chief investigator, Centre for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, clarified that getting "sufficient delivery of the therapeutic payload to the peripheral nerve" is a hurdle.
