Abstract

Our knowledge about gene regulation and function in eukaryotes considerably improved with the discovery of ribonucleic acid interference (RNAi) mechanism. This system, which has been preserved among species throughout evolution, is based on the fact that double-stranded RNAs can trigger breaking down of complementary messenger RNA sequences. Recently, these small RNA sequences have become a powerful tool for gene silencing in biological studies. Despite the relatively recent discovery of RNAi, in 1998, it has already been applied in research for therapeutical purposes. Indeed, dominant disease genes have been silenced with this method, thus RNAi can be considered as an important milestone in molecular therapy. RNAi strategies have been developed for animal models of various neurodegenerative diseases, and this technique has been used in Huntington disease (HD) models for the last five years. In this review, after explaining the mechanism of RNAi, we will discuss the developments in this field, how this method can be used in the treatment of HD and the challenges ahead.