ClinVar Miner

Submissions for variant NM_004646.4(NPHS1):c.2417C>A (p.Ala806Asp)

gnomAD frequency: 0.00001  dbSNP: rs386833912
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Total submissions: 12
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Submitter RCV SCV Clinical significance Condition Last evaluated Review status Method Comment
GeneDx RCV000712426 SCV005439249 pathogenic not provided 2024-06-19 criteria provided, single submitter clinical testing Published functional studies demonstrate that this variant impairs transport of mutated proteins to the cell surface (PMID: 11726550); Not observed at significant frequency in large population cohorts (gnomAD); This variant is associated with the following publications: (PMID: 24742477, 21415313, 32462257, 19812541, 20507940, 9915943, 11317351, 23349334, 11726550, 29474669, 33980730)
Women's Health and Genetics/Laboratory Corporation of America, LabCorp RCV000049886 SCV005381053 pathogenic Finnish congenital nephrotic syndrome 2024-08-15 criteria provided, single submitter clinical testing Variant summary: NPHS1 c.2417C>A (p.Ala806Asp) results in a non-conservative amino acid change located in the Immunoglobulin subtype 2 domain (IPR003598) of the encoded protein sequence. Three of five in-silico tools predict a damaging effect of the variant on protein function. The variant allele was found at a frequency of 8e-06 in 251454 control chromosomes. c.2417C>A has been reported in the literature in individuals affected with Nephrotic Syndrome, Type 1 (example: Lenkkeri_1999, Santin_2009, Lovric_2014). These data indicate that the variant is likely to be associated with disease. At least one publication reports experimental evidence evaluating an impact on protein function. The most pronounced variant effect impairs cellular localization (Liu_2001). The following publications have been ascertained in the context of this evaluation (PMID: 9915943, 19812541, 11726550, 24742477). ClinVar contains an entry for this variant (Variation ID: 56473). Based on the evidence outlined above, the variant was classified as pathogenic.
Baylor Genetics RCV000049886 SCV004191424 pathogenic Finnish congenital nephrotic syndrome 2024-02-15 criteria provided, single submitter clinical testing
Vasylyeva lab, Texas Tech University Health Sciences Center RCV000049886 SCV004123130 pathogenic Finnish congenital nephrotic syndrome 2020-09-16 criteria provided, single submitter clinical testing
Labcorp Genetics (formerly Invitae), Labcorp RCV000712426 SCV001385277 pathogenic not provided 2024-03-13 criteria provided, single submitter clinical testing This sequence change replaces alanine, which is neutral and non-polar, with aspartic acid, which is acidic and polar, at codon 806 of the NPHS1 protein (p.Ala806Asp). This variant is present in population databases (rs386833912, gnomAD 0.002%). This missense change has been observed in individuals with congenital nephrotic syndrome (PMID: 9915943, 20507940, 21415313, 24742477, 33980730). ClinVar contains an entry for this variant (Variation ID: 56473). Advanced modeling of protein sequence and biophysical properties (such as structural, functional, and spatial information, amino acid conservation, physicochemical variation, residue mobility, and thermodynamic stability) performed at Invitae indicates that this missense variant is expected to disrupt NPHS1 protein function with a positive predictive value of 80%. Experimental studies have shown that this missense change affects NPHS1 function (PMID: 11726550). This variant disrupts the p.Ala806 amino acid residue in NPHS1. Other variant(s) that disrupt this residue have been determined to be pathogenic (PMID: 23349334; Invitae). This suggests that this residue is clinically significant, and that variants that disrupt this residue are likely to be disease-causing. For these reasons, this variant has been classified as Pathogenic.
Illumina Laboratory Services, Illumina RCV000049886 SCV000915827 pathogenic Finnish congenital nephrotic syndrome 2018-08-22 criteria provided, single submitter clinical testing The NPHS1 c.2417C>A (p.Ala806Asp) variant has been reported in at least five studies and is found in a homozygous state in three probands and in a compound heterozygous state in two probands with congenital Finnish nephrosis (Lenkerri et al. 1999; Santin et al. 2009; Santin et al. 2011; Lovric et al. 2014; Machuca et al. 2014; Berody et al. 2018). The p.Ala806Asp variant was absent from 30 controls and is reported at a frequency of 0.00002 in the European (non-Finnish) population of the Exome Aggregation Consortium but this is based on one allele so the variant is presumed to be rare. Immunostaining of cells with wildtype NPHS1 demonstrated expression at the plasma membrane, whereas in cells containing the p.Ala806Asp variant there was no detectable surface staining observed (Liu et al. 2001). Based on the evidence, the p.Ala806Asp variant is classified as pathogenic for congenital nephrotic syndrome. This variant was observed by ICSL as part of a predisposition screen in an ostensibly healthy population.
Athena Diagnostics RCV000712426 SCV000842919 pathogenic not provided 2019-12-30 criteria provided, single submitter clinical testing The frequency of this variant in the general population is consistent with pathogenicity. Conflicting predictions of the effect on the protein. In multiple individuals, this variant has been seen with a single recessive pathogenic variant in the same gene, suggesting this variant may also be pathogenic. Assessment of experimental evidence suggests this variant results in abnormal protein function.
Equipe Genetique des Anomalies du Developpement, Université de Bourgogne RCV000049886 SCV000803816 pathogenic Finnish congenital nephrotic syndrome 2016-06-07 criteria provided, single submitter clinical testing
Counsyl RCV000049886 SCV000220399 likely pathogenic Finnish congenital nephrotic syndrome 2014-06-12 criteria provided, single submitter literature only
Solve-RD Consortium RCV000049886 SCV005091303 likely pathogenic Finnish congenital nephrotic syndrome 2022-06-01 no assertion criteria provided provider interpretation Variant confirmed as disease-causing by referring clinical team
Natera, Inc. RCV000049886 SCV001460528 pathogenic Finnish congenital nephrotic syndrome 2020-09-16 no assertion criteria provided clinical testing
Juha Muilu Group; Institute for Molecular Medicine Finland (FIMM) RCV000049886 SCV000082295 likely pathogenic Finnish congenital nephrotic syndrome no assertion criteria provided not provided Converted during submission from probable-pathogenic to Likely pathogenic.

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