SiRNA therapeutic design: Tools and challenges

Amanda P. Malefyt, Phillip A. Angart, Christina Chan, S. Patrick Walton

Research output: Chapter in Book/Report/Conference proceedingChapter

  • 3 Citations

Abstract

Current RNA-based therapeutics are principally focused toward activating the RNA interference (RNAi) pathway through exogenous administration of short interfering RNAs (siRNAs) and sometimes short hairpin RNAs (shRNAs). The promise of RNAi-based therapeutics arises from their broad applicability and excellent specificity. This chapter reviews siRNA design strategies for improving intracellular interactions with the RNAi pathway proteins as well as key characteristics required for the design of optimal delivery vehicles to maximize specific silencing in only the cells of interest. The status of previous and ongoing clinical trials will be described as these provide insight for overcoming future challenges for long-term use of RNAi as a therapeutic modality.

Original languageEnglish (US)
Title of host publicationRegulatory RNAs: Basics, Methods and Applications
PublisherSpringer-Verlag Berlin Heidelberg
Pages475-503
Number of pages29
ISBN (Print)9783642225178, 3642225160, 9783642225161
DOIs
StatePublished - Aug 1 2013

Profile

RNA Interference
Small Interfering RNA
Clinical Trials
RNA
Proteins
Analgesia

Keywords

  • Asymmetry
  • clinical trials
  • immune response
  • nanoparticles
  • nanotechnology
  • nucleic acid delivery
  • off-target effects
  • RNAi
  • siRNA
  • therapeutics

ASJC Scopus subject areas

  • Biochemistry, Genetics and Molecular Biology(all)

Cite this

Malefyt, A. P., Angart, P. A., Chan, C., & Walton, S. P. (2013). SiRNA therapeutic design: Tools and challenges. In Regulatory RNAs: Basics, Methods and Applications (pp. 475-503). Springer-Verlag Berlin Heidelberg. DOI: 10.1007/978-3-642-22517-8_19

SiRNA therapeutic design : Tools and challenges. / Malefyt, Amanda P.; Angart, Phillip A.; Chan, Christina; Walton, S. Patrick.

Regulatory RNAs: Basics, Methods and Applications. Springer-Verlag Berlin Heidelberg, 2013. p. 475-503.

Research output: Chapter in Book/Report/Conference proceedingChapter

Malefyt, AP, Angart, PA, Chan, C & Walton, SP 2013, SiRNA therapeutic design: Tools and challenges. in Regulatory RNAs: Basics, Methods and Applications. Springer-Verlag Berlin Heidelberg, pp. 475-503. DOI: 10.1007/978-3-642-22517-8_19
Malefyt AP, Angart PA, Chan C, Walton SP. SiRNA therapeutic design: Tools and challenges. In Regulatory RNAs: Basics, Methods and Applications. Springer-Verlag Berlin Heidelberg. 2013. p. 475-503. Available from, DOI: 10.1007/978-3-642-22517-8_19

Malefyt, Amanda P.; Angart, Phillip A.; Chan, Christina; Walton, S. Patrick / SiRNA therapeutic design : Tools and challenges.

Regulatory RNAs: Basics, Methods and Applications. Springer-Verlag Berlin Heidelberg, 2013. p. 475-503.

Research output: Chapter in Book/Report/Conference proceedingChapter

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