Both adeno-associated virus (AAV2) vector expressing HTT-silencing micro RNA (miRNA) [69] and intra-ventricular delivered antisense oligonucleotides (ASO) [70] were able to reduce the level of Htt mRNA and protein and to determine an amelioration on motor and behavioural features in different mouse (YAC128, BACHD, R6/2), and non human primate models of HD. The gene discussed is HTT; the disease is Huntington disease.