MicroRNA-based therapeutic strategies for cancer

MicroRNAs (miRNAs) with a role in cancer can act either as oncogenic miRNAs (oncomiRs), which are upregulated in the tumor or as tumor suppressor miRNAs, which are downregulated. These alterations are responsible for the deregulation of the pathways relevant to cancer. Therefore different approaches have been proposed to inhibit oncomiRs or restore the expression of tumor suppressor miRNAs, as a way to restore the cellular equilibrium. Such efforts have demonstrated the potential of miRNA-based therapeutic approaches. However, optimizing the delivery of miRNA-based therapeutics is a major concern since an adequate level of the therapeutic is needed in tumor site, while minimizing off-target effects and toxicities is a must. In this chapter, we will summarize the recent progress in miRNA-based therapeutic strategies for cancer and highlight the challenges and breakthroughs of such strategies, and we will show the state-of-the-art of miRNA-based therapeutics in three different malignancies: pancreatic cancer, multiple myeloma, and non–small cell lung cancer.