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Methods to Identify and Characterize siRNAs Targeting Serpin A1 In Vitro and In Vivo Using RNA Interference

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Alpha-1 Antitrypsin Deficiency

Part of the book series: Methods in Molecular Biology ((MIMB,volume 1639))

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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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Authors and Affiliations

  1. Alnylam Pharmaceuticals, 300 Third St, Cambridge, MA, 02155, USA

    Stuart Milstein, Kun Qian, Linying Zhang & Alfica Sehgal

Authors
  1. Stuart Milstein
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  2. Kun Qian
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  3. Linying Zhang
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  4. Alfica Sehgal
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Corresponding author

Correspondence to Alfica Sehgal .

Editor information

Editors and Affiliations

  1. Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester, Massachusetts, USA

    Florie Borel

  2. Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester, Massachusetts, USA

    Christian Mueller

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Milstein, S., Qian, K., Zhang, L., Sehgal, A. (2017). Methods to Identify and Characterize siRNAs Targeting Serpin A1 In Vitro and In Vivo Using RNA Interference. In: Borel, F., Mueller, C. (eds) Alpha-1 Antitrypsin Deficiency . Methods in Molecular Biology, vol 1639. Humana Press, New York, NY. https://doi.org/10.1007/978-1-4939-7163-3_11

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  • DOI: https://doi.org/10.1007/978-1-4939-7163-3_11

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  • Publisher Name: Humana Press, New York, NY

  • Print ISBN: 978-1-4939-7161-9

  • Online ISBN: 978-1-4939-7163-3

  • eBook Packages: Springer Protocols

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