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In Vivo Production of Small Interfering RNAs That Mediate Gene Silencing

*Abstract: Nobel laureate and Howard Hughes Medical Investigator Dr. Craig Mello and Howard Hughes Medical Investigator Dr. Phil Zamore and colleagues have engineered novel artificial RNA precursors for use in gene silencing in vivo. These novel stem-loop RNA molecules are designed using endogenous miRNA precursors called pre-miRNAs, as templates. Like premiRNAs, their short duplex nature does not activate the IFN response pathway, avoiding unwanted side effects. In addition, because they are based on endogenous precursor molecules, they are recognized and processed by the naturally occurring RNAi processing enzyme Dicer. Unlike pre-miRNA molecules, they contain complementarity to the target mRNA molecule and Dicer processes them into functional siRNA molecules. These functional siRNA molecules result in gene silencing via RISC-mediated cleavage of the target mRNA. In addition, regulatory sequences are operably linked to the precursor molecules allowing for temporal and spatial regulation in vivo. These novel RNA molecules are applicable in cell culture and non-human animals in the form of a vector substrate or transgene. These artificial precursors are ideal for in vivo gene therapy because they are based on naturally occurring gene silencing molecules and are processed accordingly, providing minimum side-effects with maximum gene silencing.

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