MiRNAs are a class of small, naturally occurring RNA molecules that play critical roles in modulating numerous biological pathways by regulating gene expression. The knowledge that miRNA expression is dysregulated in many pathological disease processes, including cancer, has led to a rapidly expanding body of literature as we try to unveil their mechanism of action. Their putative role as oncogenes or tumour suppressor genes presents a wonderful opportunity to provide targeted cancer treatment strategies. Additionally, their documented function in a host of benign diseases broadens the potential market for miRNA-based therapeutics. The present review outlines the underlying rationales for considering mi(cro)RNAs as therapeutic agents or targets. We highlight the potential of manipulating miRNAs for the treatment of many common diseases, particularly cancers. Finally, we summarize the challenges that need to be overcome to fully harness the potential of miRNA-based therapies so they become the next generation of pharmaceutical products.