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
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ATLANTA (June 21, 2023) CMT Research Foundation (CMTRF), a patient-led, non-profit focused on delivering treatments and cures for Charcot-Marie-Tooth disease*, today announced that Dr. William W. Motley, a neurologist with a doctorate in neurogenetics with fifteen...
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DTx Pharma
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DTx Pharma Video – Using siRNA to Treat Charcot-Marie-Tooth Disease Type 1A
Thomas F. Sander Joins CMT Research Foundation Board of Directors
ATLANTA (April 26, 2023) CMT Research Foundation (CMTRF), a patient-led, non-profit focused on delivering treatments and cures for Charcot-Marie-Tooth disease, today announced that rare disease sales specialist, Thomas F. Sander, has joined its Board of Directors....
Edritz Javelosa to Join the CMT Research Foundation as Chief Scientific Officer
ATLANTA (April 17, 2023) Edritz Javelosa (Hav-a-low-sa), PhD, who has been at the Muscular Dystrophy Association (MDA) since 2019 and Research Portfolio Director since 2021, has joined the CMT Research Foundation (CMTRF) as Chief Scientific Officer. Reporting to...
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