However, in Huntington’s disease (HD), like in all trinucleotide disorders of the CNS, the classical dogma purports that the abnormal protein, in this case mutant huntingtin (mHtt), causes cellular dysfunction through a cell-autonomous manner that results in aggregation, inclusion body formation and cell death [8, 13, 19, 23, 24, 48]. The gene discussed is HTT; the disease is Huntington disease.