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Trends Mol Med. 2016 Jan;22(1):28-37. doi: 10.1016/j.molmed.2015.11.005. Epub 2015 Dec 14.

Small Molecule Modulators of Pre-mRNA Splicing in Cancer Therapy.

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National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA; Department of Biochemistry and Molecular Biology, the Institute for Medical Research Israel-Canada, Hebrew University-Hadassah Medical School, Jerusalem 91120, Israel. Electronic address:
National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA. Electronic address:


Pre-mRNA splicing is a fundamental process in mammalian gene expression and alternative RNA splicing plays a considerable role in generating protein diversity. RNA splicing events are also key to the pathology of numerous diseases, particularly cancers. Some tumors are molecularly addicted to specific RNA splicing isoforms making interference with pre-mRNA processing a viable therapeutic strategy. Several RNA splicing modulators have recently been characterized, some showing promise in preclinical studies. While the targets of most splicing modulators are constitutive RNA processing components, possibly leading to undesirable side effects, selectivity for individual splicing events has been observed. Given the high prevalence of splicing defects in cancer, small molecule modulators of RNA processing represent a potentially promising novel therapeutic strategy in cancer treatment. Here, we review their reported effects, mechanisms, and limitations.


cancer therapy; splicing modulators

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