Vivo production of small interfering RNAs that mediate gene silencing


Ontology type: sgo:Patent     


Patent Info

DATE

2013-09-10T00:00

AUTHORS

Phillip D. Zamore , Juanita McLachlan , Gyorgy Hutvagner , Alla Grishok , Craig C. Mello

ABSTRACT

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 vivo 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. More... »

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