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Updated: Oct 3, 2025

Use of Ultra-high Field MRI in Small Rodent Models of Polycystic Kidney Disease for In Vivo Phenotyping and Drug Monitoring
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AMPK and Polycystic Kidney Disease Drug Development: An Interesting Off-Target Target

Michael J Caplan1

  • 1Department of Cellular and Molecular Physiology, Yale University School of Medicine, New Haven, CT, United States.

Frontiers in Medicine
|February 17, 2022
PubMed

Insights

Autosomal Dominant Polycystic Kidney Disease (ADPKD) disrupts kidney structure and cellular signaling. Targeting cellular energy pathways, specifically adenosine monophosphate-stimulated protein kinase, may offer new therapeutic strategies for ADPKD.

Area of Science:

  • Nephrology
  • Genetics
  • Cellular Biology

Background:

  • Autosomal Dominant Polycystic Kidney Disease (ADPKD) is a genetic disorder characterized by significant alterations in kidney structure and cellular signaling pathways.
  • The precise mechanisms linking ADPKD-causing gene mutations to these signaling disruptions are not fully understood.
  • Altered cellular metabolism is a hallmark of ADPKD.

Purpose of the Study:

  • To investigate the role of cellular metabolism in ADPKD.
  • To explore the involvement of adenosine monophosphate-stimulated protein kinase (AMPK) in ADPKD.
  • To evaluate AMPK modulation as a potential therapeutic strategy for ADPKD.

Main Methods:

  • Analysis of cellular metabolism in ADPKD models.
  • Assessment of AMPK activity in affected cells.
  • Investigation of therapeutic potential of AMPK modulators.

Main Results:

  • Cellular metabolism is significantly altered in ADPKD.
  • AMPK, a key regulator of cellular energy, shows perturbed activity in ADPKD cells.
  • Modulating AMPK activity shows promise in preclinical studies.

Conclusions:

  • AMPK plays a critical role in cellular energy regulation within the context of ADPKD.
  • Targeting AMPK presents a promising avenue for developing novel ADPKD therapeutics.
  • Further research into AMPK modulation could lead to effective treatments for ADPKD.
Keywords:
Autosomal Dominant Polycystic Kidney DiseaseCFTRadenosine monophosphate-stimulated protein kinasemTORmetabolismmetformin

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