Total submissions: 2
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Gene |
RCV003443388 | SCV004168332 | likely pathogenic | not provided | 2023-04-24 | criteria provided, single submitter | clinical testing | Reported in a patient with L-dopa responsive dystonia in published literature (Steinberger et al., 2000); Intronic +5 splice site variant in a gene for which loss of function is a known mechanism of disease, and splice predictors support a deleterious effect; Not observed at significant frequency in large population cohorts (gnomAD); This variant is associated with the following publications: (PMID: 25525159, 11113234) |
Labcorp Genetics |
RCV003778486 | SCV004570945 | uncertain significance | Dystonia 5; GTP cyclohydrolase I deficiency | 2023-07-06 | criteria provided, single submitter | clinical testing | 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. This sequence change falls in intron 5 of the GCH1 gene. It does not directly change the encoded amino acid sequence of the GCH1 protein. It affects a nucleotide within the consensus splice site. This variant is not present in population databases (gnomAD no frequency). This variant has been observed in individual(s) with dystonia (PMID: 11113234). Variants that disrupt the consensus splice site are a relatively common cause of aberrant splicing (PMID: 17576681, 9536098). Algorithms developed to predict the effect of sequence changes on RNA splicing suggest that this variant may disrupt the consensus splice site. |