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Targeted RNA Degradation Allows Precision Repurposing of Protein-Targeted Small Molecule Medicine to RNA

  • Repurposing of medicines has potential to accelerate treatments to patients
  • RNA is considered nearly impossible to target directly with small molecules
  • Dovitinib,  targets receptor tyrosine kinases (RTKs) and  binds to a functional site in the oncogenic microRNA precursor, pre-miR-21, inhibiting its processing
  • A RIBOTAC of Dovitinib shifted the selectivity to pre-miR-21 by 2500-fold
  • Targeted degradation affected the progression of  metastasis of breast cancer and Alport syndrome, a genetically defined disease of the kidney
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