IN VIVO PRODUCTION OF SMALL INTERFERING RNAS THAT MEDIATE GENE SILENCING

摘要

The invention provides engineered RNA precursors that when expressed in a ce ll are processed by the cell to produce targeted small interfering RNAs (siRNAs ) that selectively silence targeted genes (by cleaning specific mRNAs) using t he cell's own RNA interference (RNAi) pathway. By introducing nucleic acid molecules that encode these engineered RNA precursors into cells in vivo wit h appropriate regulatory sequences, expression of the engineered RNA precursor s can be selectively controlled both temporally and spatially, i.e., at particular times and/or in particular tissues, organs, or cells.