• Nat Rev Drug Discov · Nov 2013

    Review

    MicroRNAs and other non-coding RNAs as targets for anticancer drug development.

    • Hui Ling, Muller Fabbri, and George A Calin.
    • 1] Experimental Therapeutics and Leukemia Department, MD Anderson Cancer Center, University of Texas, Houston, Texas 77030, USA. [2].
    • Nat Rev Drug Discov. 2013 Nov 1;12(11):847-65.

    AbstractThe first cancer-targeted microRNA (miRNA) drug - MRX34, a liposome-based miR-34 mimic - entered Phase I clinical trials in patients with advanced hepatocellular carcinoma in April 2013, and miRNA therapeutics are attracting special attention from both academia and biotechnology companies. Although miRNAs are the most studied non-coding RNAs (ncRNAs) to date, the importance of long non-coding RNAs (lncRNAs) is increasingly being recognized. Here, we summarize the roles of miRNAs and lncRNAs in cancer, with a focus on the recently identified novel mechanisms of action, and discuss the current strategies in designing ncRNA-targeting therapeutics, as well as the associated challenges.

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