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Updated: May 31, 2026

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
Polycystic kidney disease: inheritance, pathophysiology, prognosis, and treatment
Christian R Halvorson1, Matthew S Bremmer, Stephen C Jacobs
1Department of Surgery, University of Maryland School of Medicine, Baltimore, MD, USA. chalv001@umaryland.edu
Abstract:
Both autosomal dominant and recessive polycystic kidney disease are conditions with severe associated morbidity and mortality. Recent advances in the understanding of the genetic and molecular pathogenesis of both ADPKD and ARPKD have resulted in new, targeted therapies designed to disrupt cell signaling pathways responsible for the abnormal cell proliferation, dedifferentiation, apoptosis, and fluid secretion characteristic of the disease. Herein we review the current understanding of the pathophysiology of these conditions, as well as the current treatments derived from our understanding of the mechanisms of these diseases.
Insights
Autosomal dominant and recessive polycystic kidney disease (ADPKD and ARPKD) cause significant health problems. New therapies target the genetic and molecular causes of these kidney diseases, addressing abnormal cell growth and fluid secretion.
Area of Science:
- Nephrology
- Genetics
- Molecular Biology
Background:
- Autosomal dominant polycystic kidney disease (ADPKD) and autosomal recessive polycystic kidney disease (ARPKD) are significant genetic kidney disorders.
- These conditions are characterized by severe morbidity and mortality, impacting numerous patients worldwide.
Purpose of the Study:
- To review the current understanding of the pathophysiology of ADPKD and ARPKD.
- To discuss novel, targeted therapies developed based on recent advances in genetic and molecular pathogenesis.
Main Methods:
- Literature review of recent advances in polycystic kidney disease research.
- Analysis of genetic and molecular mechanisms underlying ADPKD and ARPKD.
- Evaluation of current and emerging therapeutic strategies.
Main Results:
- Understanding the genetic and molecular basis of ADPKD and ARPKD has led to targeted therapies.
- These therapies aim to disrupt specific cell signaling pathways involved in disease progression.
- Key pathways targeted include those regulating cell proliferation, dedifferentiation, apoptosis, and fluid secretion.
Conclusions:
- Recent insights into polycystic kidney disease pathogenesis have enabled the development of targeted treatments.
- These novel therapies offer potential to mitigate the severe morbidity and mortality associated with ADPKD and ARPKD.
- Continued research into disease mechanisms is crucial for further therapeutic advancements.
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