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Cystic fibrosis (CF), an autosomal recessive disorder, significantly affects the function of exocrine glands. This genetically inherited disease is characterized by the production of thick and sticky mucus, which can severely affect various organs and systems in the body.
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Reduced decay-accelerating factor expression promotes complement-mediated cystogenesis in murine ADPKD.

Sofia Bin1,2,3, Miran Yoo4, Paolo Molinari1

  • 1Translational Transplant Research Center and Department of Medicine, Icahn School of Medicine at Mount Sinai, New York, New York, USA.

JCI Insight
|June 24, 2024
PubMed
Summary
This summary is machine-generated.

Loss of Pkd1 in autosomal dominant polycystic kidney disease (ADPKD) promotes complement activation, driving cyst growth. Inhibiting C5aR1 offers a potential therapeutic target for ADPKD.

Keywords:
ComplementNephrology

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Area of Science:

  • Nephrology
  • Immunology
  • Genetics

Background:

  • Autosomal dominant polycystic kidney disease (ADPKD) is a genetic disorder caused by mutations in PKD1 or PKD2.
  • Patients exhibit complement activation in renal fluids, but its role in cyst development is unknown.
  • PKD1 deficiency is linked to altered complement gene expression and reduced complement regulators.

Purpose of the Study:

  • To investigate the causal link between complement activation and cyst growth in ADPKD.
  • To explore the role of complement component 3 (C3) and its receptors in ADPKD pathogenesis.
  • To identify potential therapeutic targets for ADPKD by examining complement pathways.

Main Methods:

  • Utilized a Pkd1-deficient (Pkd1KO) renal tubular cell line.
  • Generated conditional Pkd1-/- C3-/- mice and Pkd1-/- C3+/+ controls.
  • Assessed gene and protein expression of complement factors and regulators; evaluated cystogenesis, renal function, and inflammation.

Main Results:

  • Pkd1-deficient cells showed increased complement gene expression (C3, C5, C3aR, C5aR1) and decreased complement regulators (DAF, CD59, Crry).
  • Pkd1-/- C3-/- mice exhibited reduced cystogenesis, preserved kidney function, and less inflammation compared to controls.
  • Restoring Pkd1 function or inhibiting C5aR1 significantly reduced cell proliferation in Pkd1KO cells.

Conclusions:

  • Loss of Pkd1 in renal tubular cells leads to uncontrolled complement activation via downregulation of DAF.
  • Enhanced C5a formation and C5aR1 activation promote ADPKD cyst growth.
  • C5aR1 represents a promising therapeutic target for managing ADPKD.