RNA SILENCING COMPOSITIONS AND METHODS FOR THE TREATMENT OF HUNTINGTON'S DISEASE

摘要

The present invention relates to the discovery of an effective treatment for a variety of Huntington's disease (HD). The present invention utilizes RNA silencing technology (e.g. RNAi) against single nucleotide polymorphisms (SNPs) in the Huntingtin (htt) gene encoding the dominant, gain-of-function mutant Huntington protein, thereby resulting in an effective treatment for the gain-of-function disease.