TRIPARTITE RNAI CONSTRUCTS

The present invention provides compositions and methods for inhibiting expression of a target gene in a cell. The process comprises introduction of double-stranded tripartite RNAi constructs into the cells and reducing the expression of the corresponding messenger RNA in the cells. The constructs, w...

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Bibliographische Detailangaben
Hauptverfasser: SAMARSKY, DMITRY, SALOMON, WILLIAM, WAGNER, RICK, USMAN, NASSIM, PAVCO, PAM, WOOLF, TOD M
Format: Patent
Sprache:eng ; fre
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Beschreibung
Zusammenfassung:The present invention provides compositions and methods for inhibiting expression of a target gene in a cell. The process comprises introduction of double-stranded tripartite RNAi constructs into the cells and reducing the expression of the corresponding messenger RNA in the cells. The constructs, which may be packaged in or delivered as sequestered RNAi constructs, differ from the canonical siRNA in that they comprise a tripartite structure which follows the general formula of having (1) an RNAi core (either native or abbreviated), (2) one or more terminal moieties attached to the RNAi core and optionally (3) a linker between the RNAi core and the terminal moiety. Once packaged into sequestration vehicles, the constructs are activated for gene regulation by the application of certain forms of energy.