After years of intensive research following the discovery of abnormally expanded polyQ (> 36Q) in huntingtin exon 1 (Httex1) as the cause for HD [3], mutant Htt (mHtt) protein was found to alter the protein structure and properties and cause the protein aggregation leading to the dysregulation of many cellular processes, such as gene transcription, proteostasis, mitochondrial function and chromatin modification, resulting in cortico-striatal miscommunication and progressive neuronal loss [1, 4–6]. The gene discussed is HTT; the disease is Huntington disease.