MicroRNAs As Promising Therapeutic Targets

MicroRNAs (miRNA) represent a class of noncoding RNAs that regulate gene expression by binding and inhibiting mRNA expression. Various human disorders, such as cancer, cardiovascular diseases, bone diseases, autoimmune diseases, and inflammatory diseases, are influenced by numerous deregulated miRNAs, which inhibit genes necessary for normal phenotypes or promote the expression of those that are normally suppressed. This influence, in turn, determines the fate of the cells and disease progression. Many are either upregulated or downregulated in the disease state, and their levels in serum can be used to diagnose the state or progression of the disorder, allowing them to be targeted either by inhibitors or mimics, respectively. In some situations, the same miRNA is expressed in various diseased tissues and even displays targeting of the same mRNA or more than one mRNA. The advancements in chemical modifications and targeted delivery systems have paved the way for miRNAs to be used in clinical trials to establish their therapeutic potential. In this chapter, we have summarized various strategies that can be employed to target miRNAs in diverse diseases and the current and completed clinical trials involving miRNAs.