DUAL FUNCTIONAL OLIGONUCLEOTIDES FOR USE IN REPRESSING MUTANT GENE EXPRESSION

摘要

The present invention is based, in part, on the discovery that endogenous miRNAs can be recruited for translational repression of target mRNAs. The RNA-silencing agents and the methods described herein, thereby provide a means by which to treat genetic (e.g., genetic neurodegenerative diseases such as Huntington's Disease) or non-genetic diseases by, for example, blocking the synthesis of proteins that contribute to the diseases. Accordingly the RNA-silencing agents of the present invention have an mRNA targeting moiety, a linking moiety, and an miRNA recruiting moiety.