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.
New Evidence for Gene Replacement in a Mouse Model of Charcot-Marie-Tooth Disease
Important Research News Announcements
- Genomic Studies in Charcot-Marie-Tooth Disease July 31, 2024
- Charcot-Marie-tooth disease type 2A: An update on pathogenesis and therapeutic perspectives March 5, 2024
- Accelerate Clinical Trials in CMT (ACTCMT) Study March 1, 2024
- Creation of a Schwann Cell Gene Regulatory Network February 29, 2024
- Activation of XBP1s attenuates disease severity in models of proteotoxic Charcot-Marie-Tooth type 1B [Preprint] February 2, 2024