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RNAi as a Tool for Mammalian Gene Analysis: Applications of siRNAs

Down-Syndrome cell-adhesion molecule (12). In each of the screening applications, the keys to the experiments have been robust phenotypic assays and high quality RNAi libraries.


siRNAs as Therapeutics: The Next Frontier
While many researchers are exploiting siRNAs in their drug development processes, some scientists are evaluating siRNAs as therapeutic agents (reviewed in 13). If realized, siRNAs could make it possible to target virtually any gene for therapeutic intervention. Researchers have already shown that the RNAi pathway is active in mice and that siRNAs are tolerated and effective in several different tissues (14). Synthetic siRNAs and siRNA expression vectors (both plasmid and viral) have been injected systemically and into defined tissues and elicited target-specific responses. A number of publications have shown that siRNAs can inhibit the replication HIV (15,16) and Hepatitis B (17). Additionally, an siRNA targeting a prion-prone protein was capable of inhibiting prion formation in cells, creating an alternative therapeutic approach to prion diseases (18).

As the RNAi field continues to develop, moving into animal models, therapeutics, and drug discovery and targeting, Ambion will continue to develop innovative products that harness the power of RNAi for applications to basic, applied and therapeutic research efforts.


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