The DOI matches its Crossref record; 18 of the 18 references it cites were found in a registry or an index.
Knocking down disease: a progress report on siRNA therapeutics
Open accessAbstract
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.
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Cites
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Cited by
Therapeutic siRNA: state of the art
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Tau-targeting therapies for Alzheimer disease
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In vitro and ex vivo strategies for intracellular delivery
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Metadata
- Authors
- Wittrup, A., & Lieberman, J.
- Venue
- Nature Reviews Genetics
- Published
- 2015-08-18
- Volume
- 16
- Issue
- 9
- Pages
- 543-552
- ISSN
- 1471-0056 (ISSN-L)
- 1471-0064
- OpenAlex
- W2120820989
- Cited by
- 804
- References
- 18
- FWCI
- 6.74
- Type
- journal article
- Language
- en
- Open access
- green
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