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Updated: Dec 8, 2025

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Autophagy induction promotes renal cyst growth in polycystic kidney disease

Eun Ji Lee1, Je Yeong Ko1, Sumin Oh1

  • 1Department of Biological Science, Sookmyung Women's University, Seoul 04310, Republic of Korea.

Ebiomedicine
|September 19, 2020
PubMed
Abstract

Insights

Autophagy regulation does not alleviate polycystic kidney disease (PKD). However, autophagy inhibitors show promise for treating specific autosomal dominant polycystic kidney disease (ADPKD) patients, warranting cautious consideration of these drugs.

Area of Science:

  • Nephrology
  • Cell Biology
  • Molecular Medicine

Background:

  • Polycystic kidney disease (PKD) is characterized by renal cyst formation from proliferating tubular cells.
  • Autophagy is a cellular process implicated in PKD pathogenesis and is a potential therapeutic target.
  • Mammalian target of rapamycin (mTOR) is a critical regulator of autophagy, but its role in PKD cystogenesis remains unclear.

Purpose of the Study:

  • To investigate the effect of autophagy regulation on cystogenesis in polycystic kidney disease (PKD).
  • To evaluate the therapeutic potential of modulating autophagy in preclinical models of PKD.

Main Methods:

  • Clinical validation using GEO datasets and autosomal dominant polycystic kidney disease (ADPKD) patient samples.
  • In vivo studies utilizing newly established PKD and LC3 transgenic mice.
  • In vitro and in vivo experiments with various autophagy drugs.

Main Results:

  • Autophagy stimulation and LC3 overexpression did not alleviate PKD.
  • An autophagy inhibitor demonstrated an inhibitory effect on cyst development.
  • These findings suggest a potential therapeutic application for autophagy inhibitors in a subset of ADPKD patients.

Conclusions:

  • Autophagy modulation offers a novel perspective on PKD pathogenesis.
  • Drugs targeting autophagy regulation should be approached with caution in PKD treatment strategies.
  • Further research is needed to elucidate the precise role of autophagy in PKD and to identify patient subgroups that may benefit from specific autophagy-modulating therapies.
Keywords:
AutophagyIft46PKDPolycystic kidney

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