RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING MULTIFUNCTIONAL SHORT INTERFERING NUCLEIC ACID (MULTIFUNCTIONAL SINA)

摘要

[00101] The present invention concerns methods and nucleic acid based reagentsuseful in modulating gene expression in a variety of applications, includinguse in therapeutic, veterinary, agricultural, diagnostic, target validation,and genomic discovery applications. Specifically, the invention relates tomultifunctional short interfering nucleic acid (multifunctional siNA)molecules that modulate the expression of one or more genes in a biologicsystem, such as a cell, tissue, or organism via RNA interference (RNAi). Thebifuntional short interfering nucleic acid (multifunctional siNA) molecules ofthe invention can target more than one regions of nucleic acid sequence in asingle target nucleic acid molecule or can target regions of nucleic acidsequence in differing target nucleic acid molecules. The self multifunctionalsiNA molecules are useful in the treatment of any disease or condition thatresponds to modulation of gene expression or activity in a cell, tissue, ororganism.