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.

Related News & Research

Walking Manhattan to Find a Cure

From a young age, Rivka knew that there was something different about her compared to her three siblings; that difference was Charcot-Marie-Tooth (CMT) disease.Meet Rivka Ligier, a CMT1B patient who is preparing to participate in this year's Great Saunter, a one-day,...

Vanderbilt’s Charles Sanders Successfully Finds Molecules That Alter PMP22 Production or Cell Surface Trafficking; In Next Phase Will Test if They Can Improve CMT-like Problems in Schwann Cells

CMT1A is caused by a gene-copying event that results in the overproduction of the peripheral myelin protein 22 (PMP22) in Schwann cells. The excess PMP22 protein fails to traffic normally to the cell surface and instead collects inside the cells as clumps of proteins...

CMT Research Update/Armatus Bio

Armatus Bio has partnered with Andelyn Biosciences, Inc., a cell and gene therapy Contract Development and Manufacturing Organization to accelerate manufacturing of an Armatus gene therapy treatment for Charcot-Marie-Tooth Type 1A. The goal is to maximize program...


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