Dysregulation of MicroRNA Expression and Human Diseases?

Abstract

Data from in vivo gain- and loss-of-function studies in mouse models demonstrate without a doubt that microRNAs (miRNAs), alongside coding genes, control diverse biological processes in mammalians. The regulation of miRNA expression is tightly controlled, and often the same rules and regulations that govern coding gene expression apply also to miRNAs. Similar to coding genes, altering the levels and the temporal expression of a specific miRNA clearly affects the proper development and function of the tissue where it is expressed. Therefore, it is reasonable to argue that the dysregulated control of miRNA expression would give rise to diseases as has been well established for coding genes. Along the same line of reasoning, there is an impetus to use miRNAs as diagnostic biomarkers and to develop therapeutic agents to target miRNAs for the treatment of various diseases. In this chapter, we will summarize studies establishing the physiological roles of miRNAs in mammalians and discuss whether there is a link between dysregulated miRNA expression and diseases. In addition, we will review progresses toward targeting miRNAs as therapeutic agents.