Total submissions: 4
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Invitae | RCV002526993 | SCV003441189 | uncertain significance | not provided | 2022-04-15 | criteria provided, single submitter | clinical testing | This sequence change replaces tyrosine, which is neutral and polar, with cysteine, which is neutral and slightly polar, at codon 343 of the FLI1 protein (p.Tyr343Cys). This variant is not present in population databases (gnomAD no frequency). This missense change has been observed in individual(s) with clinical features of FLI1-related conditions (PMID: 24100448). ClinVar contains an entry for this variant (Variation ID: 424633). Algorithms developed to predict the effect of missense changes on protein structure and function (SIFT, PolyPhen-2, Align-GVGD) all suggest that this variant is likely to be disruptive. Experimental studies have shown that this missense change affects FLI1 function (PMID: 24100448). In summary, the available evidence is currently insufficient to determine the role of this variant in disease. Therefore, it has been classified as a Variant of Uncertain Significance. |
Gene |
RCV002526993 | SCV003923805 | likely pathogenic | not provided | 2023-03-03 | criteria provided, single submitter | clinical testing | Published functional studies suggest a damaging effect (luciferase assays revealed transcriptional activity of Y343C was significantly reduced compared to wildtype) (Stockley et al., 2013); Not observed at significant frequency in large population cohorts (gnomAD); 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: 24100448, 27479822, 35021601) |
OMIM | RCV000487469 | SCV000574548 | pathogenic | Bleeding disorder, platelet-type, 21 | 2017-04-25 | no assertion criteria provided | literature only | |
ISTH- |
RCV000487469 | SCV002515571 | likely pathogenic | Bleeding disorder, platelet-type, 21 | no assertion criteria provided | research |