Design of siRNA therapeutics from the molecular scale

Phillip Angart, Daniel Vocelle, Christina Chan, S. Patrick Walton

Research output: Contribution to journalArticle

  • 15 Citations

Abstract

While protein-based therapeutics is well-established in the market, development of nucleic acid therapeutics has lagged. Short interfering RNAs (siRNAs) represent an exciting new direction for the pharmaceutical industry. These small, chemically synthesized RNAs can knock down the expression of target genes through the use of a native eukaryotic pathway called RNA interference (RNAi). Though siRNAs are routinely used in research studies of eukaryotic biological processes, transitioning the technology to the clinic has proven challenging. Early efforts to design an siRNA therapeutic have demonstrated the difficulties in generating a highly-active siRNA with good specificity and a delivery vehicle that can protect the siRNA as it is transported to a specific tissue. In this review article, we discuss design considerations for siRNA therapeutics, identifying criteria for choosing therapeutic targets, producing highly-active siRNA sequences, and designing an optimized delivery vehicle. Taken together, these design considerations provide logical guidelines for generating novel siRNA therapeutics.

LanguageEnglish (US)
Pages440-468
Number of pages29
JournalPharmaceuticals
Volume6
Issue number4
DOIs
StatePublished - Mar 25 2013

Profile

Small Interfering RNA
Therapeutics
Biological Phenomena
Drug Industry
RNA Interference
Nucleic Acids
Guidelines
RNA
Technology
Gene Expression
Research
Proteins

Keywords

  • Delivery vehicle design
  • Liver cancer
  • RNAi
  • siRNA design
  • siRNA therapeutic

ASJC Scopus subject areas

  • Molecular Medicine
  • Pharmaceutical Science

Cite this

Design of siRNA therapeutics from the molecular scale. / Angart, Phillip; Vocelle, Daniel; Chan, Christina; Patrick Walton, S.

In: Pharmaceuticals, Vol. 6, No. 4, 25.03.2013, p. 440-468.

Research output: Contribution to journalArticle

Angart, Phillip ; Vocelle, Daniel ; Chan, Christina ; Patrick Walton, S./ Design of siRNA therapeutics from the molecular scale. In: Pharmaceuticals. 2013 ; Vol. 6, No. 4. pp. 440-468
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