MicroRNAs and polycystic kidney disease

Lama Noureddine, Sachin Hajarnis, Vishal Patel

Research output: Contribution to journalArticle

11 Scopus citations


Polycystic kidney disease (PKD), the most common genetic cause of chronic renal failure, is characterized by the presence of numerous fluid-filled cysts in renal parenchyma. Despite recent progress, no FDA-approved therapy is available to retard cyst growth. Here, we review current evidence implicating two groups of microRNAs (miRNAs) - the miR-17∼92 cluster and miR-200s - in the pathogenesis of PKD. We present a new hypothesis for cyst growth involving miRNAs and regulation of PKD gene dosage. We propose that manipulating miRNA function in an attempt to normalize PKD gene dosage represents a novel therapeutic strategy in PKD.

Original languageEnglish (US)
JournalDrug Discovery Today: Disease Models
Issue number3
Publication statusPublished - 2013


ASJC Scopus subject areas

  • Drug Discovery
  • Molecular Medicine

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