MicroRNAs and Polycystic Kidney Disease
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ABSTRACT
MicroRNAs (miRNAs) are a class of small non protein-coding RNAs that function as inhibitors of post-transcriptional gene expression in plants and animals. Over a thousand different miRNAs are known to be encoded by the human genome, the majority of which are conserved in other species. miRNAs are essential for virtually all aspects of mammalian biology, including development of key organs such the brain, the heart, and the kidney. More importantly, miRNAs are implicated in the pathogenesis of numerous common human diseases, and pharmaceutical manipulation of miRNA function has emerged as an exciting new therapeutic approach for cancer and kidney diseases. Several lines of evidence have connected miRNAs to the pathogenesis of polycystic kidney disease (PKD). miRNAs are aberrantly expressed in cystic kidneys and this aberrant expression is thought to regulate key aspects of cyst pathogenesis such as cyst epithelial cell proliferation and apoptosis as well as dosage of the various cystic kidney disease genes. In this chapter, we briefly discuss the basic biology of miRNAs and their role in kidney development, and highlight the role of three miRNA families – miR-17 and related miRNAs, miR-200 family and miR-21- in the pathogenesis of PKD.
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