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Nucleic Acid Ther. 2019 Apr;29(2):61-66. doi: 10.1089/nat.2018.0762. Epub 2018 Dec 18.

RNA Interference-Based Cancer Drugs: The Roadblocks, and the "Delivery" of the Promise.

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Division of Pharmacoengineering and Molecular Pharmaceutics, Center for Nanotechnology in Drug Delivery, Eshelman School of Pharmacy, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina.


Nucleic acid-based therapeutics like synthetic small interfering RNAs have been exploited to modulate gene function, taking advantage of RNA interference (RNAi), an evolutionally conserved biological process. Recently, the world's first RNAi drug was approved for a rare genetic disorder in the liver. However, there are significant challenges that need to be resolved before RNAi can be translated in other genetic diseases like cancer. Current drug delivery platforms for therapeutic silencing RNAs are tailored to hepatic targets. RNAi therapies for nonhepatic conditions are still at early clinical phases. In this study, we discuss the critical design considerations in anticancer RNAi drug development, insights gained from initial clinical trials, and new strategies that are entering clinical development, shaping the future of RNAi in cancer.


RNA interference; cancer therapy; gene silencing; siRNA

[Available on 2020-04-01]

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