FP042ATMIN MODULATES PKHD1 EXPRESSION AND THROUGH ALTERED NON-CANONICAL WNT/PLANAR CELL POLARITY (PCP) SIGNALLING MEDIATES ARPKD SEVERITY

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Tissue-specific regulation of the mouse Pkhd1 (ARPKD) gene promoter.

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Multi-exon deletions of the PKHD1 gene cause autosomal recessive polycystic kidney disease (ARPKD).

BACKGROUND Autosomal recessive polycystic kidney disease (ARPKD) is caused by mutations in the PKHD1 (polycystic kidney and hepatic disease 1) gene on chromosome 6p12, a large gene spanning 470 kb of genomic DNA. So far, only micromutations in the 66 exons encoding the longest open reading frame (ORF) have been described, and account for about 80% of mutations. OBJECTIVE To test the hypothesi...

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LETTER TO JMG Comprehensive genomic analysis of PKHD1 mutations in ARPKD cohorts

A utosomal recessive polycystic kidney disease (ARPKD; MIM 263200) is an important childhood nephropathy, occurring in 1 in 20 000 live births. The clinical phenotype is dominated by dilatation of the renal collecting ducts, biliary dysgenesis, and portal tract fibrosis. Affected children often present in utero with enlarged, echogenic kidneys, as well as oligohydramnios secondary to poor urine...

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ژورنال

عنوان ژورنال: Nephrology Dialysis Transplantation

سال: 2018

ISSN: 0931-0509,1460-2385

DOI: 10.1093/ndt/gfy104.fp042