CMT1X

CMT1X – Dr. Rudolph Martini at the University of Würzburg, Germany, has found that inhibiting the macrophages associated with inflammation has a very positive effect in a mouse model of CMT1X, which is caused by mutation of the GJB1 gene. Based on his studies, we are developing approaches to inhibit macrophages as a clinical treatment. In addition, the work of Dr. Kleopas Kleopa has shown the first example of a successful gene therapy in a CMT1X mouse model, and he is continuing these studies to optimize this novel type of therapy for not only CMT1X but also CMT4.