Overview of Gene Silencing by RNA Interference

Daniel H. Kim1, John J. Rossi2

1 Howard Hughes Medical Institute, Department of Molecular Biology, Massachusetts General Hospital, and Department of Genetics, Harvard Medical School, Boston, Massachusetts, 2 Graduate School of Biological Sciences and Division of Molecular Biology, Beckman Research Institute of the City of Hope, Duarte, California
Publication Name:  Current Protocols in Nucleic Acid Chemistry
Unit Number:  Unit 16.1
DOI:  10.1002/0471142700.nc1601s36
Online Posting Date:  March, 2009
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Abstract

The potential of harnessing RNA interference (RNAi) for sequence‐specific gene silencing has generated much excitement and progress in the field. Recent advances in RNAi technology suggest that RNAi‐based approaches may soon become an effective therapeutic strategy against a myriad of diseases. This overview provides a brief description of important considerations when designing an RNAi‐based method for gene silencing and therapeutic development: (a) mechanistic aspects of RNAi‐mediated gene silencing in mammalian cells; (b) structural requirements for potent siRNA duplexes; (c) off‐target effects and interferon responses; and (d) effective delivery of RNAi‐inducing agents. Promising therapeutic applications of RNAi that are currently in the developmental pipeline are also described. Curr. Protoc. Nucleic Acid Chem. 36:16.1.1‐16.1.10. © 2009 by John Wiley & Sons, Inc.

Keywords: RNAi; siRNA; microRNA; shRNA; transcriptional gene silencing

     
 
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Table of Contents

  • Introduction
  • RNAi Mechanisms
  • Enhancing RNAi Potency
  • Improving RNAi Specificity
  • Delivering RNA
  • Therapeutic RNAi Applications
  • Conclusions
  • Acknowledgements
  • Literature Cited
  • Figures
     
 
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Materials

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Literature Cited

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