Total submissions: 9
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Counsyl | RCV000169004 | SCV000220144 | likely pathogenic | Nephropathic cystinosis | 2014-03-03 | criteria provided, single submitter | literature only | |
Center for Pediatric Genomic Medicine, |
RCV000514820 | SCV000609716 | likely pathogenic | not provided | 2017-05-22 | criteria provided, single submitter | clinical testing | |
Fulgent Genetics, |
RCV000763395 | SCV000894116 | likely pathogenic | Ocular cystinosis; Juvenile nephropathic cystinosis; Nephropathic cystinosis | 2022-04-08 | criteria provided, single submitter | clinical testing | |
Labcorp Genetics |
RCV002512766 | SCV000935698 | pathogenic | Ocular cystinosis; Juvenile nephropathic cystinosis; Inborn genetic diseases | 2024-01-08 | criteria provided, single submitter | clinical testing | This sequence change replaces glycine, which is neutral and non-polar, with arginine, which is basic and polar, at codon 197 of the CTNS protein (p.Gly197Arg). This variant is present in population databases (rs113994207, gnomAD 0.03%). This missense change has been observed in individual(s) with CTNS-related conditions (PMID: 10625078, 11505338, 28649545). In at least one individual the data is consistent with being in trans (on the opposite chromosome) from a pathogenic variant. ClinVar contains an entry for this variant (Variation ID: 4451). 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 CTNS protein function with a positive predictive value of 80%. Experimental studies have shown that this missense change affects CTNS function (PMID: 15128704, 29467429). Algorithms developed to predict the effect of sequence changes on RNA splicing suggest that this variant may create or strengthen a splice site. For these reasons, this variant has been classified as Pathogenic. |
Women's Health and Genetics/Laboratory Corporation of America, |
RCV001844005 | SCV002103764 | pathogenic | Cystinosis | 2022-02-14 | criteria provided, single submitter | clinical testing | Variant summary: CTNS c.589G>A (p.Gly197Arg) results in a non-conservative amino acid change in the encoded protein sequence. Four of five in-silico tools predict a damaging effect of the variant on protein function. The variant allele was found at a frequency of 5.6e-05 in 251352 control chromosomes (gnomAD). This frequency is not higher than expected for a pathogenic variant in CTNS causing Cystinosis (5.6e-05 vs 0.0025), allowing no conclusion about variant significance. c.589G>A has been reported in the literature in multiple compound heterozygous individuals affected with Cystinosis (e.g. Anikster_2000, Kleta_2001, Phornphutkul_2001). These data indicate that the variant is very likely to be associated with disease. Experimental evidence evaluating an impact on protein function demonstrated the variant confers low level of cystine transport and also, leads to a significant trafficking defect and is found to go to the cell surface (Kalatzis_2004, Deshpande_2018). Three ClinVar submitters (evaluation after 2014) cite the variant as pathogenic/likely pathogenic. Based on the evidence outlined above, the variant was classified as pathogenic. |
Baylor Genetics | RCV000169004 | SCV004212944 | pathogenic | Nephropathic cystinosis | 2024-03-30 | criteria provided, single submitter | clinical testing | |
Juno Genomics, |
RCV000763395 | SCV005418355 | pathogenic | Ocular cystinosis; Juvenile nephropathic cystinosis; Nephropathic cystinosis | criteria provided, single submitter | clinical testing | PM2_Supporting+PS3_Supporting+PM3_VeryStrong+PP4 | |
OMIM | RCV000004704 | SCV000024879 | pathogenic | Ocular cystinosis | 2000-01-01 | no assertion criteria provided | literature only | |
Gene |
RCV000004704 | SCV000041135 | not provided | Ocular cystinosis | no assertion provided | literature only |