Author(s): Ramiro Garzon, Guido Marcucci, Carlo M Croce
MicroRNAs (miRNAs) are evolutionary conserved small non-coding RNAs that regulate gene expression. Early studies have shown that miRNA expression is deregulated in cancer, and experimental data indicate that cancer phenotypes can be modified by targeting miRNA expression. Based on these observations, miRNA-based anticancer therapies are being developed either alone or in combination with current targeted therapies, with the goal to improve disease response and increase cure rate. The advantage of using miRNA approaches is based on the ability to concurrently target multiple effectors of pathways involved in cell differentiation, proliferation and survival. In this review, we describe the role of miRNAs in tumorigenesis, and critically discuss the rationale, strategies and challenges for therapeutic targeting of miRNAs in cancer.