By: Keith N. Fargo, Ph.D., Chief Scientific Officer, CMT Research Foundation
The CMT Research Foundation is currently funding a research project led by DTx Pharma to design genetic therapies for Charcot-Marie-Tooth disease (CMT). By attaching the genetic sequences to molecules called long chain fatty acids, they allow the therapies to target cells in the nerves at much lower concentrations than traditional approaches. This is important in gene therapy because higher concentrations can cause dangerous immune system reactions. Although this research is still in preliminary stages — only being tested in cells and animals at this time — this approach, if successful, may overcome a fundamental hurdle in using gene therapies for peripheral nerve disease.
Most recently, the scientists at DTx Pharma have identified several genetic sequences that reduce the levels of PMP22 in animal models of CMT1A. One in particular was especially effective at lowering PMP22 and showed no signs of an immune response. Further testing and refinement is now underway to select the best candidates to take forward in development. Additionally, the researchers at DTx have been awarded a grant from the National Institutes of Health that effectively quadruples the initial CMT Research Foundation investment.
Read more about previous updates from this project:
We will continue to keep you updated on the latest progress from this study. In the meantime, learn more about our research priorities and see all the CMT research we’re currently funding. You can also sign up to be the first to receive email updates about new research and scientific progress.
Help Advance CMT Drug Development
While no treatments or cures currently exist for CMT, the science to change that does.
Your donation to the CMT Research Foundation will fund cutting-edge research that’s solely focused on drug development and continue moving promising projects like the one at DTx Pharma forward without delay. Together, we will change the course of CMT during our lifetime — and for every future generation.