Investigation of axonal transport deficits in Charcot-Marie-Tooth disease type 1A and modification through selective inhibition of histone deacetylase 6 KU Leuven
Charcot-Marie-Tooth disease (CMT) is the most common inherited neurological disorder of the peripheral nervous system (PNS). Patients suffer from mild-to-severe muscle loss at the most distal regions of their body in a ‘stock-glove’ distribution. Duplications in the gene coding for peripheral myelin protein 22 (PMP22) causes the most common form of CMT, CMT type 1A (CMT1A). PMP22 is an essential protein for the initiation and maintenance of ...