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Methods to Identify and Characterize siRNAs Targeting Serpin A1 In Vitro and In Vivo Using RNA Interference
Stuart Milstein1, Kun Qian1, Linying Zhang1
1Alnylam Pharmaceuticals, 300 Third St, Cambridge, MA, 02155, USA.
Methods in Molecular Biology (Clifton, N.J.)
|July 29, 2017
Abstract:
RNAi is a powerful tool that can be used to probe gene function as well as for therapeutic intervention. We describe a workflow and methods to identify screen and select potent and specific siRNAs in vitro and in vivo using qPCR-based methods as well as an AAT activity assay. We apply these techniques to a set of siRNAs targeting rat AAT, and use this set to exemplify the cell-based and in vivo data that can be generated using these methods.
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