Year 3

Overall we have made excellent progress on this proposal. We have successfully generated iPSC lines from CMT1A patients and characterized them; validated our TALEN approach to genetically “correct” the mutation in CMT1A patient iPSC lines; established differentiation protocols for iPSC lines into neural crest stem cells and tested several methods to generated Schwann cell precursors; imported and validated a rat model of CMT1A to be used for intraneural stem cell injection experiments.