In CMT1B, the myelin sheath of the peripheral nervous system is compromised because of a dysfunctional protein created by mutations within a gene called, myelin protein zero, or MPZ (aka P0), a necessary component of the myelin sheath. With funding from the CMT research foundation, Dr. Rashnonejad is creating a gene therapy that has the potential to treat all or nearly all forms of CMT1B. The research team aims to design a gene therapy that both reduces the expression of the mutant MPZ and replaces it with healthy MPZ. Dr. Rashnonejad has already designed these elements of the gene therapy. The next step, made possible by your generosity, is to further develop them, package them together in an AAV9 delivery vector, and test the full therapeutic for safety and efficacy in CMT1B mouse models.