Vanderbilt School of Medicine Basic Sciences

CMT1A is known to be caused by overproduction of PMP22 protein. Early research indicates that excess PMP22 protein within the cell interferes with transport of the protein to the cell surface, causing toxicities, and leading to defects in the myelin sheath.  Normalizing PMP22’s transport to the cell surface could benefit CMT1A symptoms. This project will utilize Vanderbilt University’s capabilities to screen a collection of small molecule compounds for their activity to improve PMP22 transport and normalization of myelin formation in CMT1A mouse neurons.  Completion of these studies has potential to impact discovery of a novel therapeutic approach for the treatment of CMT1A.