Therapeutic delivery of microRNAs

08-24-2017

Therapeutic delivery of microRNAs in the absence of toxic delivery vehicles: Work from the Kasinski and Low labs has identified a novel mechanism capable of delivering therapeutic microRNAs in the absence of toxicity. Historically, microRNAs have been packaged inside of protective delivery vehicles in order to shield the microRNA from serum nucleases. Unfortunately, the added benefit of protecting the microRNA comes with the risk of toxicity associated with the vehicle. In a recent report published in Science Translational Medicine, first author Esteban Orellana removed the vehicle entirely and showed that conjugating a microRNA directly to a targeting ligand, in this case folate leads to rapid and specific tumor uptake resulting in efficacy for both breast and lung cancers. Importantly, using this novel chemistry there were no signs of toxicity even when the microRNA was administered at doses greater than 100-fold over the therapeutic dose. Other Purdue authors that contributed to this work include Srinivasarao Tenneti (currently at Ohio State), Loganathan Rangasmy, and Tiffany Lyle.

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