Related Experiment Video

Updated: Mar 23, 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

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ALDH1A1 is a potential novel target for treatment of ADPKD

Liwen Zhang1, John Cijiang He2

  • 1Department of Medicine/Nephrology, Icahn School of Medicine at Mount Sinai, New York, New York, USA; Department of Nephrology, Zhongshan Hospital, Fudan University, Shanghai, China.

Kidney International
|March 21, 2026
PubMed

Insights

Aldehyde dehydrogenase 1A1 is a novel risk factor for autosomal dominant polycystic kidney disease (ADPKD). Targeting this enzyme with disulfiram and immune checkpoint inhibitors shows promise for treating ADPKD.

Area of Science:

  • Nephrology
  • Molecular Biology
  • Pharmacology

Background:

  • Autosomal dominant polycystic kidney disease (ADPKD) is a common genetic disorder.
  • Identifying novel therapeutic targets is crucial for managing ADPKD progression.

Purpose of the Study:

  • To investigate the role of aldehyde dehydrogenase 1A1 (ALDH1A1) in ADPKD pathogenesis.
  • To evaluate the therapeutic potential of ALDH1A1 inhibition and immune modulation in ADPKD.

Main Methods:

  • Identification of ALDH1A1 as a risk factor in ADPKD.
  • In vivo studies using ADPKD mouse models treated with disulfiram (ALDH1A1 inhibitor) and anti-programmed death ligand 1 (PD-L1) antibody.
  • Assessment of cyst growth, proliferative pathways, and immune microenvironment.

Main Results:

  • ALDH1A1 acts as a transcription factor, activating proliferative pathways in ADPKD.
  • Disulfiram treatment significantly delayed cyst growth in ADPKD mice.
  • Combination therapy with disulfiram and anti-PD-L1 antibody synergistically reduced cyst progression and improved the renal immune microenvironment.

Conclusions:

  • ALDH1A1 is a key driver of ADPKD and a potential therapeutic target.
  • Repurposing ALDH1A1 inhibitors and PD-L1 blockades offers a novel therapeutic strategy for ADPKD.
  • Further research into ALDH1A1's transcriptional mechanisms in kidney disease is warranted.

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