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Updated: May 12, 2026

Use of Ultra-high Field MRI in Small Rodent Models of Polycystic Kidney Disease for In Vivo Phenotyping and Drug Monitoring
07:35

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: new horizons and therapeutic frontiers

P G Czarnecki1, T I Steinman

  • 1Division of Nephrology and Brigham & Women's Hospital, Renal Division and Harvard Medical School, Department of Medicine, Beth Israel Deaconess Medical Center, Boston, MA, USA.

Minerva Urologica E Nefrologica = the Italian Journal of Urology and Nephrology
|March 30, 2013
PubMed

Insights

Autosomal dominant polycystic kidney disease (ADPKD) is a common inherited kidney disorder. Research is uncovering signaling pathways involved in cyst development, aiming for targeted therapies beyond symptom management.

Area of Science:

  • Nephrology
  • Genetics
  • Molecular Biology

Background:

  • Autosomal dominant polycystic kidney disease (ADPKD) is the most common inherited kidney disease, causing significant renal and systemic morbidity.
  • Despite the identification of Pkd-1 and Pkd-2 genes, the precise molecular mechanisms underlying ADPKD pathogenesis remain unclear.
  • Current ADPKD management focuses on alleviating chronic kidney disease symptoms such as hypertension, anemia, hyperparathyroidism, and pain, as specific therapies are lacking.

Purpose of the Study:

  • To review the current understanding of molecular mechanisms in ADPKD pathogenesis.
  • To identify potential therapeutic targets based on signaling pathways involved in cystogenesis.
  • To summarize the limited clinical evidence for existing and emerging ADPKD treatments.

Main Methods:

  • Review of intensive research on ADPKD and related cystic kidney diseases.
  • Analysis of intracellular signaling pathways, including ciliary function, calcium, and cAMP.
  • Evaluation of proliferative and tissue patterning responses involving mTOR, STAT, CDK, and wnt signaling.
  • Assessment of clinical evidence from randomized controlled trials and ongoing studies.

Main Results:

  • Intracellular signaling pathways dependent on ciliary function, including calcium and cAMP mechanisms, are implicated in ADPKD.
  • mTOR, STAT, CDK, and wnt signaling pathways play crucial roles in cystogenesis and represent potential therapeutic targets.
  • Clinical trials for tolvaptan are ongoing, while everolimus and sirolimus have shown limited benefit in slowing PKD progression.

Conclusions:

  • Understanding the complex signaling networks in ADPKD is critical for developing targeted therapies.
  • Future research will focus on small molecule inhibitors of growth factor receptor, CDK, and STAT pathways.
  • Development of novel biomarkers for disease progression and therapeutic response is essential for advancing ADPKD treatment.

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