A clinical study revealed that intrathecal administration of antisense oligonucleotides (ASO) can decrease the levels of mutant huntingtin protein (mHTT) in the cerebrospinal fluid of Huntington's disease (HD) patients, and other studies also have demonstrated that silencing HTT mRNA can effectively rescue various animal models of HD [22, 26, 31, 32]. The gene discussed is HTT; the disease is Huntington disease.