In an article published yesterday in Neurology Today, Dr. Kleopa demonstrated that gene replacement could be a viable treatment for recessive forms of CMT. CMT type 4C is a demyelinating disease, and this treatment both decreased the number of unmyelinated fibers and increased the thickness of myelin in mouse models of the disease. In their studies, the treated mice performed better in functional tests than mice which were untreated. Further testing will include the use of viral vectors, a virus delivery mechanism that is also being explored for CMT1A, also a demyelinating disease, by Dr. Kleopa in work funded by the CMTRF.
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