miR-34 - a microRNA replacement therapy is headed to the clinic

Front Genet. 2012 Jul 2;3:120. doi: 10.3389/fgene.2012.00120. eCollection 2012.


MicroRNA-34 (miR-34) is a master regulator of tumor suppression. It is downregulated in numerous cancers and inhibits malignant growth by repressing genes involved in various oncogenic signaling pathways. Consequently, miR-34 antagonizes processes that are necessary for basic cancer cell viability as well as cancer stemness, metastasis, and chemoresistance. This broad anti-oncogenic activity holds the prospect of creating a new remedy that is effective against tumor heterogeneity. This review focuses on the molecular mechanisms of miR-34-mediated tumor suppression, pharmacologies in animal models of cancer, and a status update of a miR-34 therapy that may be among the first miRNA mimics to reach the clinic.

Keywords: miR-34; miR-34a; miRNA; miRNA replacement; miRNA therapeutics.