Three congruent human Schwann cell models of CMT1A reveal a converged phenotype

Three CMT1A patient Schwann cell populations which were derived from hESC, hiPSC, or direct converted hiNC share the disease relevant phenotypes. From comparing deep sequencing results of these populations, potential therapeutic targets were newly identified. Schwann cells from CMT1A hESC, hiPSC, or hfibroblast were analyzed for RNA sequencing followed by gene ontology analysis. We were able to purify discrete Schwann cell populations at Schwann cell precursor or neural crest stages by using Schwann cell surface marker and FACS sorting. We differentiated human Schwann cell precursors from hESCs (H9 or PGD CMT1A hESCs) and hiPSCs (healthy donor 00024, 02623, and CMT1A disease line 05148, 05165) by using 2i protocol, and WNT was activated for iNC differentiation.