Article
Neuromuscular Dysfunction and Charcot-Marie-Tooth Disease Reversal in Mfn2 T105M Knock-In Rats.
International journal of molecular sciences - 18 Aug 2026
Weigele Jochen, Franco Antonietta, Dorn Gerald W
Abstract excerpt
Charcot-Marie-Tooth (CMT) disease type 2A is a rare heritable disorder caused by pathogenic variants of mitofusin (MFN) 2 that suppress mitochondrial fusion and motility in peripheral nerves, culminating in denervation myoatrophy. The rarity of this condition and the limited choice of animal models preclude pre-clinical evaluation of many tests that could be translated to human trials. Here, we introduced the...
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