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Literature record

A novel mutation in NPHS2 causing nephrotic syndrome in a Saudi Arabian family.

PMID 25949463 | PMCID PMC4421432 | DOI 10.1093/ndtplus/sfq149 · NDT plus · 2010

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We report a consanguineous family from Saudi Arabia with three affected children presenting with infantile nephrotic syndrome. In order to provide a molecular diagnosis, a genome-wide SNP analysis of the affected patients was performed. We identified a region of homozygosity on chromosome 1, containing the NPHS2 gene. Direct sequencing, by exon PCR, of NPHS2 identified a homozygous nucleotide change 385C > T within exon 3 in the three affected children, leading to a premature stop codon (Q129X). This homozygous truncating mutation in NPHS2 is novel and was associated with a severe clinical phenotype. Additional mutations in related genes NPHS1, PLCE1 and NEPH1 were not identified, excluding tri-allelism within these genes in this family.

Validated evidence

TypeEntitySource evidenceConfidenceExtractor
geneNPHS2“A novel mutation in NPHS2 causing nephrotic syndrome in a Saudi Arabian family.”0.98hgnc_dict_v1
geneNPHS1“Additional mutations in related genes NPHS1, PLCE1 and NEPH1 were not identified, excluding tri-allelism within these genes in this family.”0.98hgnc_dict_v1
genePLCE1“Additional mutations in related genes NPHS1, PLCE1 and NEPH1 were not identified, excluding tri-allelism within these genes in this family.”0.98hgnc_dict_v1
phenotypenephrotic syndrome“A novel mutation in NPHS2 causing nephrotic syndrome in a Saudi Arabian family.”0.98phenotype_alias_lexicon_v2
populationSaudi Arabia“A novel mutation in NPHS2 causing nephrotic syndrome in a Saudi Arabian family. We report a consanguineous family from Saudi Arabia with three affected children presenting with infantile nephrotic syndrome. Direct sequencing, by exon PCR, of NPHS2 identified a homozygous nucleotide change 385C > T within exon 3 in the three affected children, leading to a premature stop codon (Q129X).”0.95saudi_context_rules_v1
variantQ129X“Direct sequencing, by exon PCR, of NPHS2 identified a homozygous nucleotide change 385C > T within exon 3 in the three affected children, leading to a premature stop codon (Q129X).”0.82literature_variant_regex_v2