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The promises and pitfalls of RNA-interference-based therapeutics

Author

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  • Daniela Castanotto

    (Beckman Research Institute of the City of Hope)

  • John J. Rossi

    (Beckman Research Institute of the City of Hope)

Abstract

The discovery that gene expression can be controlled by the Watson–Crick base-pairing of small RNAs with messenger RNAs containing complementary sequence — a process known as RNA interference — has markedly advanced our understanding of eukaryotic gene regulation and function. The ability of short RNA sequences to modulate gene expression has provided a powerful tool with which to study gene function and is set to revolutionize the treatment of disease. Remarkably, despite being just one decade from its discovery, the phenomenon is already being used therapeutically in human clinical trials, and biotechnology companies that focus on RNA-interference-based therapeutics are already publicly traded.

Suggested Citation

  • Daniela Castanotto & John J. Rossi, 2009. "The promises and pitfalls of RNA-interference-based therapeutics," Nature, Nature, vol. 457(7228), pages 426-433, January.
    • Handle: RePEc:nat:nature:v:457:y:2009:i:7228:d:10.1038_nature07758
    DOI: 10.1038/nature07758
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    Cited by:

    1. Changzheng Li & Weijun Fu & Yu Zhang & Liang Zhou & Zhi Mao & Weiran Lv & Juan Li & Ye Zhou, 2015. "Meta-Analysis of MicroRNA-146a rs2910164 G>C Polymorphism Association with Autoimmune Diseases Susceptibility, an Update Based on 24 Studies," PLOS ONE, Public Library of Science, vol. 10(4), pages 1-12, April.

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