4.5 Review

New approaches to target RNA binding proteins

Journal

CURRENT OPINION IN CHEMICAL BIOLOGY
Volume 62, Issue -, Pages 13-23

Publisher

ELSEVIER SCI LTD
DOI: 10.1016/j.cbpa.2020.12.006

Keywords

RNA binding proteins (RBPs); High throughput screening (HTS); Chemical probes

Funding

  1. DOD-Advanced Research Projects Agency [DARPA D19AP00041]
  2. NIH NIGMS [T32 GM136614]

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RBPs are a diverse class of proteins that regulate RNA biology and their dysregulation is implicated in various diseases. Discovery of small molecules targeting RBPs has sparked interest in new strategies for chemical probe discovery in this protein class. Advance in high throughput screening technologies is expected to unlock the full therapeutic potential of RBPs.
RNA binding proteins (RBPs) are a large and diverse class of proteins that regulate all aspects of RNA biology. As RBP dysregulation has been implicated in a number of human disorders, including cancers and neurodegenerative disease, small molecule chemical probes that target individual RBPs represent useful tools for deciphering RBP function and guiding the production of new therapeutics. While RBPs are often thought of as tough-to-drug, the discovery of a number of small molecules that target RBPs has spurred considerable recent interest in new strategies for RBP chemical probe discovery. Here we review current and emerging technologies for high throughput RBP-small molecule screening that we expect will help unlock the full therapeutic potential of this exciting protein class.

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