The regulative role of DAXX in the gene expression in C9HRE ALS/FTD patient motor neurons

To investigate the role of DAXX in the C9HRE-associated gene dysregulation inALS/FTD patient-derived motor neurons. Overall design: Two pairs of C9HRE ALS/FTD patient iPSCs and their isogenic controls, in which the C9Orf72 HRE mutation was removed, were differentiated into motor neurons. RNA-seq was employed to profile the global gene expression in the isogenic iMNs, C9HRE iMNs, and the C9HRE iMNs treated with shRNA targeting DAXX.