Design of siRNA therapeutics from the molecular scale

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

    Research output: Contribution to journalArticle

    • 13 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.

    Original languageEnglish (US)
    Pages (from-to)440-468
    Number of pages29
    JournalPharmaceuticals
    Volume6
    Issue number4
    DOIs
    StatePublished - Mar 25 2013

    Profile

    Small Interfering RNA
    Drug Industry
    RNA Interference
    Biological Processes
    Nucleic Acids
    RNA
    Technology
    Gene Expression
    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, Vol. 6, No. 4, 25.03.2013, p. 440-468.

    Research output: Contribution to journalArticle

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