Knocking down disease: a progress report on siRNA therapeutics
By Anders Wittrup, J. Lieberman. Nature reviews genetics, 2015
731 citations recorded by the source corpus.
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.
Subfields: Cancer Research, Molecular Biology
View Paper: https://www.semanticscholar.org/paper/78f27bc243792f8e4f28c0c0f7528081f4145b2b
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In Cancer Research
In Molecular Biology
Citations in the corpus
- Therapeutic siRNA: state of the art (2020, background): “This active targeting strategy not only reduces siRNA accumulation in unintended tissues, thus decreasing or avoiding undesired side effects and toxicity, but also achieves potent gene silencing at a low dosage.(104) siRNA is ~7–8 nm in length and 2–3 nm in diameter.”
- The delivery of therapeutic oligonucleotides (2016, background): “Because of this, much of the work with siRNA-LNPs has focused on liver diseases including transthyrethrin-mediated amyloidosis, clotting disorders, liver cancer and disorders of lipid metabolism (11,166).”