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

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NATURE REVIEWS DRUG DISCOVERY
卷 12, 期 11, 页码 847-865

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NATURE PUBLISHING GROUP
DOI: 10.1038/nrd4140

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资金

  1. University of Texas MD Anderson Research Trust
  2. University of Texas System Regents Research Scholar
  3. CLL Global Research Foundation
  4. US National Institutes of Health (NIH)/US National Cancer Institute (NCI) [CA135444]
  5. US Department of Defense Breast Cancer Idea Award
  6. Developmental Research Awards in breast cancer, ovarian cancer, brain cancer, prostate cancer, multiple myeloma and leukaemia [P50 CA100632]
  7. Specialized Programs of Research Excellence (SPOREs) [P50 CA097007]
  8. Sister Institution Network Fund (SINF) grant from the MD Anderson Cancer Center
  9. German Cancer Research Center (DKFZ) in chronic lymphocytic leukaemia
  10. SINF grant in colorectal cancer
  11. Laura and John Arnold Foundation
  12. RGK Foundation
  13. Jean Perkins Foundation
  14. Nautica Malibu Triathlon Funds
  15. Pablove Foundation
  16. St. Baldrick's Foundation
  17. Southern California Clinical and Translational Science Institute
  18. Funds from the Saban Research Institute
  19. NCI [P30CA014089]
  20. Hugh and Audy Lou Colvin Foundation
  21. T.J. Martell Foundation

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The 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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