Isolated nucleic acid molecule, vector, host cell, transgene, engineered rna precursor, non-human transgenic animal, and method of inducing ribonucleic acid interference from a target gene in a cell

摘要

"ISOLATED NUCLEIC ACID MOLECULE, VECTOR, HOST CELL, TRANSGENE, ENGINEERED RNA PRECURSOR, NON-HUMAN TRANSGENIC ANIMAL, AND METHOD OF INDUCING THE INTERFERENCE OF AN ALBIC ACID OF A CIBLE GENE The invention provides engineered RNA precursors that when expressed in a cell are processed by the cell to produce targeted small interfering RNAs (siRNAs) that selectively silence targeted genes (by clearing specific mRNAs) using its own RNA interference pathway (RNAi). cell. By introducing nucleic acid molecules encoding these engineered RNA precursors into cells in vivo with appropriate regulatory sequences, the expression of engineered RNA precursors can be selectively controlled both temporally and spatially, that is, at particular times and / or in tissues. , particular organs or cells.