Claudin-19 mutations and clinical phenotype in Spanish patients with familial hypomagnesemia with hypercalciuria and nephrocalcinosis

PLoS One. 2013;8(1):e53151. doi: 10.1371/journal.pone.0053151. Epub 2013 Jan 3.

Abstract

Familial hypomagnesemia with hypercalciuria and nephrocalcinosis is an autosomal recessive tubular disorder characterized by excessive renal magnesium and calcium excretion and chronic kidney failure. This rare disease is caused by mutations in the CLDN16 and CLDN19 genes. These genes encode the tight junction proteins claudin-16 and claudin-19, respectively, which regulate the paracellular ion reabsorption in the kidney. Patients with mutations in the CLDN19 gene also present severe visual impairment. Our goals in this study were to examine the clinical characteristics of a large cohort of Spanish patients with this disorder and to identify the disease causing mutations. We included a total of 31 patients belonging to 27 unrelated families and studied renal and ocular manifestations. We then analyzed by direct DNA sequencing the coding regions of CLDN16 and CLDN19 genes in these patients. Bioinformatic tools were used to predict the consequences of mutations. Clinical evaluation showed ocular defects in 87% of patients, including mainly myopia, nystagmus and macular colobomata. Twenty two percent of patients underwent renal transplantation and impaired renal function was observed in another 61% of patients. Results of the genetic analysis revealed CLDN19 mutations in all patients confirming the clinical diagnosis. The majority of patients exhibited the previously described p.G20D mutation. Haplotype analysis using three microsatellite markers showed a founder effect for this recurrent mutation in our cohort. We also identified four new pathogenic mutations in CLDN19, p.G122R, p.I41T, p.G75C and p.G75S. A strategy based on microsequencing was designed to facilitate the genetic diagnosis of this disease. Our data indicate that patients with CLDN19 mutations have a high risk of progression to chronic renal disease.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Child
  • Child, Preschool
  • Claudins / genetics*
  • Cohort Studies
  • Computational Biology / methods
  • DNA Mutational Analysis
  • Female
  • Genetic Predisposition to Disease*
  • Haplotypes
  • Humans
  • Hypercalciuria / complications
  • Hypercalciuria / genetics*
  • Kidney Diseases / genetics*
  • Magnesium Deficiency / complications
  • Magnesium Deficiency / genetics*
  • Male
  • Middle Aged
  • Models, Genetic
  • Nephrocalcinosis / complications
  • Nephrocalcinosis / genetics*
  • Phenotype
  • Polymorphism, Genetic
  • Spain
  • Young Adult

Substances

  • CLDN19 protein, human
  • Claudins

Grants and funding

This work was supported by research grants from Fondo de Investigación Sanitaria (FIS, PI09/91009 and PI11/00342, cofinanced by the European Regional Development Fund) and Fundación Canaria de Investigación y Salud FUNCIS (PI24/07) to FCM, and by a fellowship from the Sociedad Canaria de Pediatría de Tenerife. FS and GA acknowledge funding from FIS (grants PI09/90758 and PI09/90888, respectively). The funding from FIS was granted as coordinated proyect Renaltube. No additional external funding was received for this study. The funders have no role in study design, data collection and analysis, decision to publish or preparation of the manuscript.