Knocking down disease: a progress report on siRNA therapeutics View Full Text


Ontology type: schema:ScholarlyArticle      Open Access: True


Article Info

DATE

2015-09

AUTHORS

Anders Wittrup, Judy Lieberman

ABSTRACT

Small interfering RNAs (siRNAs), which downregulate gene expression guided by sequence complementarity, can be used therapeutically to block the synthesis of disease-causing proteins. The main obstacle to siRNA drugs - their delivery into the target cell cytosol - has been overcome to allow suppression of liver gene expression. Here, we review the results of recent clinical trials of siRNA therapeutics, which show efficient and durable gene knockdown in the liver, with signs of promising clinical outcomes and little toxicity. We also discuss the barriers to more widespread applications that target tissues besides the liver and the most promising avenues to overcome them. More... »

PAGES

543-552

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  • Identifiers

    URI

    http://scigraph.springernature.com/pub.10.1038/nrg3978

    DOI

    http://dx.doi.org/10.1038/nrg3978

    DIMENSIONS

    https://app.dimensions.ai/details/publication/pub.1007363608

    PUBMED

    https://www.ncbi.nlm.nih.gov/pubmed/26281785


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