The DAXX tax: C9orf72 DNA repeat expansions drive gain- and loss-of-function pathology in c9FTD/ALS.

ABSTRACT

In this issue of Neuron, Liu et al.1 identify DAXX as a C9orf72 hexanucleotide repeat expansion DNA-binding protein that initiates epigenetic modifications and chromatin remodeling, contributing to C9orf72 haploinsufficiency by inhibiting its stress-inducible expression and mediating both loss- and toxic gain-of-function pathology.