In vivo production of small interfering RNAs that mediate gene silencing

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 cleaving specific mRNAs) using the cell's own RNA interference (RNAi) pathway. By introducing...

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Bibliographische Detailangaben
Hauptverfasser: MCLACHLAN JUANITA, MELLO CRAIG C, GRISHOK ALLA, HUTVAGNER GYORGY, ZAMORE PHILLIP D
Format: Patent
Sprache:eng
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Zusammenfassung: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 cleaving specific mRNAs) using the cell's own RNA interference (RNAi) pathway. By introducing nucleic acid molecules that encode these engineered RNA precursors into cells in vitro with appropriate regulatory sequences, expression of the engineered RNA precursors can be selectively controlled both temporally and spatially, i.e., at particular times and/or in particular tissues, organs, or cells.