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A novel deletion in the RCA gene cluster causes atypical hemolytic uremic syndrome
Journal article   Open access   Peer reviewed

A novel deletion in the RCA gene cluster causes atypical hemolytic uremic syndrome

Tara K Maga, Nicole C Meyer, Craig Belsha, Carla J Nishimura, Yuzhou Zhang and Richard J. H Smith
Nephrology, dialysis, transplantation, Vol.26(2), pp.739-741
02/2011
DOI: 10.1093/ndt/gfq658
PMCID: PMC6937010
PMID: 20974643
url
https://doi.org/10.1093/ndt/gfq658View
Published (Version of record) Open Access

Abstract

Atypical hemolytic uremic syndrome (aHUS) is a complex complement-mediated disease that progresses to end-stage renal failure (ESRF) in 50% of cases. Dysregulation of the alternative pathway (AP) of the complement cascade manifests as microangiopathic anaemia and thrombocytopenia. Multiple genes in the AP have been implicated in disease pathogenesis. Here, we report the clinical presentation of an affected patient that was inconsistent with genotype–phenotype data for carriers of CD46 mutations. Tests of AP function in this patient suggested additional genetic factors, and in-depth studies revealed a de novo heterozygous deletion that creates a novel CFH/CFHR1 fusion protein.
aHUS Exceptional Case complement deletion kidney transplantation

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