Cell Therapies For CMT

Cell therapies involve the transplantation of human cells into an individual in order to slow or halt progression of disease. A number of different cell types can be used — including various types of stem cells. If the therapeutic cells are taken from and returned to the same individual, they are considered autologous. If they instead originate from a donor, they are allogeneic.

In some cases, a hybrid approach is used that combines elements of both cell and gene therapy. Cells may be removed from a patient, genetically modified to include a new gene, and then returned to the patient from which they were taken.

The goal in cell therapy is for the transplanted cells to counteract the effects of missing or dysfunctional proteins. With the restoration of normal protein function, it may be possible to preserve, maintain or restore physical function. 

CMT Research Foundation is leading the charge to ensure safe and effective therapies for all forms of CMT make it to the market to help those living with CMT today. We are working every day to support the families, the foundations, the academics, the scientists, the biotechs and the pharma companies who share our goal and who are working to make it happen.