ReferenceID 526

Modulation of the secretory pathway rescues zebrafish polycystic kidney disease pathology

J Am Soc Nephrol

Mutations in polycystin 1 and polycystin 2 are responsible for autosomal dominant polycystic kidney disease, the most common heritable human disease. Polycystins function as calcium ion channels, but their impact on cell

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Reference Id
526
Evidence Id
17116
Core Evidence Id
17116
Source Reference Id
1019
Herb2 Reference Id
HBREF001743
Subject Paper Key
HBIN018821_24627348
Pubmed Id
24627348
Doi
10.1681/ASN.2013101060
Paper Title
Modulation of the secretory pathway rescues zebrafish polycystic kidney disease pathology
Paper Abstract
Mutations in polycystin 1 and polycystin 2 are responsible for autosomal dominant polycystic kidney disease, the most common heritable human disease. Polycystins function as calcium ion channels, but their impact on cell physiology is not fully known. Recent findings suggest that polycystins could function in the maintenance of extracellular matrix integrity. In zebrafish, polycystin 2 knockdown induces kidney cysts, hydrocephalus, left/right asymmetry defects, and strong dorsal axis curvature. Here, we show that increased notochord sheath collagen deposition in polycystin 2-deficient embryos is directly linked to axis defects. Increased collagen II protein accumulation did not associate with increased col2a1 mRNA or a decrease in matrix metalloproteinase activity but, instead, it associated with increased expression of the endoplasmic reticulum/Golgi transport coat protein complex II Sec proteins. sec24D knockdown prevented dorsal axis curvature and kidney cystogenesis in polycystin 2 morphants. Nontoxic doses of brefeldin A also prevented the dorsal axis curvature formation in polycystin 2 morphants and curly up polycystin 2 mutants. Brefeldin A treatment after the onset of polycystin deficiency phenotypes reversed the curved axis phenotype but not kidney cyst progression. Our results suggest that polycystin 2 deficiency causes increased collagen II synthesis with upregulation of secretory pathway coat protein complex II components. Restoration of normal rates of secretory protein synthesis and secretion may be a new target in the treatment of autosomal dominant polycystic kidney disease.
Journal
J Am Soc Nephrol
Publish Year
2014
Experiment Subject
zebrafish
Experiment Type
Animal Experiment
Phenotype Related
Paper Title Cn
Paper Title En
Modulation of the secretory pathway rescues zebrafish polycystic kidney disease pathology
Bilingual Status
semi_complete