Total submissions: 3
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Prevention |
RCV003405294 | SCV004113117 | uncertain significance | NADSYN1-related disorder | 2023-03-06 | criteria provided, single submitter | clinical testing | The NADSYN1 c.395G>T variant is predicted to result in the amino acid substitution p.Trp132Leu. This variant has been reported in the compound heterozygous state in an individual with NAD deficiency (Szot et al. 2020. PubMed ID: 31883644). Functional assays in this same report showed that the p.Trp132Leu substitution causes a decrease in enzymatic activity of the protein (Szot et al. 2020. PubMed ID: 31883644). This variant is reported in 0.32% of alleles in individuals of Latino descent in gnomAD (http://gnomad.broadinstitute.org/variant/11-71175176-G-T). Although we suspect that this variant may be pathogenic, at this time, the clinical significance of this variant is uncertain due to the absence of conclusive functional and genetic evidence. |
Gene |
RCV004590090 | SCV005079612 | uncertain significance | not provided | 2023-12-04 | criteria provided, single submitter | clinical testing | Published functional studies demonstrate a damaging effect with reduced protein expression and activity (PMID: 31883644); Observed with a variant on the opposite allele (in trans) in a patient in published literature (PMID: 35491967); Observed with a variant on the opposite allele (in trans) in a fetus in published literature (PMID: 31883644); In silico analysis supports that this missense variant has a deleterious effect on protein structure/function; This variant is associated with the following publications: (PMID: 35491967, 31883644, 36951206, 37300479) |
Embryology Laboratory, |
RCV001078187 | SCV001244273 | pathogenic | Congenital NAD deficiency disorder | 2019-12-01 | no assertion criteria provided | research | This variant, c.395G>T, was found in compound heterozygosity with the pathogenic variant c.145T>C |