Year 2

During this reporting period we developed genetically corrected induced pluripotent stem cell lines from patients with CMT1A. We improved and validated a novel method for differentiating Schwann cells from iPSCs, and used this to generate human Schwann cells from patients and controls. Finally we have initiated pilot studies injecting human iPSC derived Schwann cells into the peripheral nerves of rats with myelin diseases to determine whether cell replacement therapy is a viable strategy in these disorders.