INHIBITION OF THE EXPRESSION OF HUNTINGTIN GENE

摘要

It is intended to provide a method of inhibiting the expression of a Huntington gene by using a double-stranded RNA (dsRNA); a Huntington gene expression inhibitor to be used in the inhibition of the expression of a Huntington gene as described above; and a drug for preventing and/or treatin g Huntington~s disease. Targeting to a specific mRNA sequence located in a region in the upstream vicinity of the CAG repeat of an HD gene of Huntington~s disease, the expression of the Huntington gene is inhibited wit h the use of a dsRNA that is homologous with this sequence. In this method, us e can be effectively made of a short siRNA (short double-stranded RNA) of abou t 21 to 23 bp as the dsRNA homologous to the RNA-specific sequence located in a region in the upstream vicinity of the CAG repeat. Huntington~s disease can be prevented and/or treated by administering or transferring the above-describe d dsRNA into a living mammalian body or a vital cell as a Huntington gene expression inhibitor or a preventive and/or a remedy for Huntington~s diseas e.