MicroRNAs and Polycystic Kidney Disease
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Updated: Nov 25, 2025

Use of Ultra-high Field MRI in Small Rodent Models of Polycystic Kidney Disease for In Vivo Phenotyping and Drug Monitoring
Published on: June 23, 2015
1Department of Pediatrics, Nanfang Hospital, Southern Medical University, Guangzhou, China.
Abstract:
Important advances have been made regarding the diagnosis and management of polycystic kidney diseases. Care of patients with polycystic kidney diseases has moved beyond supportive care for complications and chronic kidney disease to new potentially disease-modifying therapies. Recently, the role of noncoding RNAs, in particular microRNAs, has been described in polycystic kidney diseases. microRNAs are involved in the regulation of gene expression, in which PKD1, PKD2, and other genes that contribute to the pathogenesis of polycystic kidney diseases are considerable participants. Seminal studies have highlighted the potential importance of microRNAs as new therapeutic targets and innovative diagnostic and/or prognostic biomarkers. Furthermore, an anti-miR-17 drug has advanced through preclinical autosomal dominant polycystic disease studies, and an anti-miR-21 drug has already cleared a phase 1 clinical trial. Most probably, new drugs in the microRNA research field will be yielded as a result of ongoing and planned therapeutic trials. To provide a foundation for understanding microRNA functions as a disease-modifying therapeutic drug in novel targeted therapies, in this narrative review we present an overview of the current knowledge of microRNAs in the pathogenesis of polycystic kidney diseases.
Insights
MicroRNAs show promise in treating polycystic kidney diseases, moving beyond supportive care. These noncoding RNAs offer new therapeutic targets and diagnostic biomarkers for polycystic kidney diseases.
Area of Science:
- Biochemistry
- Genetics
- Nephrology
Background:
- Polycystic kidney diseases (PKDs) management is evolving from supportive care to disease-modifying therapies.
- Noncoding RNAs, specifically microRNAs, are increasingly recognized for their role in PKD pathogenesis.
- MicroRNAs regulate gene expression, impacting key genes like PKD1 and PKD2 involved in PKD development.
Purpose of the Study:
- To review the current understanding of microRNA functions in the pathogenesis of polycystic kidney diseases.
- To highlight the potential of microRNAs as therapeutic targets and biomarkers for PKDs.
- To provide a foundation for exploring microRNA-based disease-modifying treatments.
Main Methods:
- Narrative review of existing literature on microRNAs and polycystic kidney diseases.
- Analysis of studies investigating microRNA involvement in PKD gene regulation.
- Examination of preclinical and clinical data for microRNA-targeted therapies.
Main Results:
- MicroRNAs play a significant role in regulating genes central to PKD pathogenesis.
- Seminal studies indicate microRNAs' potential as diagnostic and prognostic biomarkers for PKDs.
- Therapeutic strategies targeting microRNAs, such as anti-miR-17 and anti-miR-21, are progressing in development.
Conclusions:
- MicroRNAs represent a promising frontier for novel targeted therapies in polycystic kidney diseases.
- Further research and clinical trials are expected to yield new microRNA-based drugs for PKDs.
- Understanding microRNA mechanisms is crucial for developing effective disease-modifying treatments for PKDs.
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