Transcriptomic changes in a mouse model of HD treated with an aggregate modulator
Huntington's disease (HD) is a hereditary movement disorder caused by a repeat expansion in the first exon of the huntingtin gene. HD is characterized by the deposition of mutant huntingtin (mHTT) aggregates, and severe neurodegeneration of the basal ganglia and neocortex. No cure is currently available and new, effective, treatment options are urgently needed. Here we study the therapeutic potetntial of a new small molecule, the oligomer modulator emrulsomin, to prevent mHTT accumulation and ameliorate HD's phenotype in the zQ175DN mouse model.