Promise of MicroRNA Replacement Therapy

Downlo roRNAs (miRNA), a class of natural RNA-interfering agents, have recently been identified as attractive s for therapeutic intervention. The rationale for developing miRNA therapeutics is based on the premise berrantly expressed miRNAs play key roles in the development of human disease, and that correcting miRNA deficiencies by either antagonizing or restoring miRNA function may provide a therapeutic benlthough miRNA antagonists are conceptually similar to other inhibitory therapies, restoring the function iRNA by miRNA replacement is a less well characterized approach. Here, we discuss the specific propof a m erties of miRNA replacement and review recent examples that explored the therapeutic delivery of miRNA mimics in animal models of cancer. Cancer Res; 70(18); 7027–30. ©2010 AACR.