Total submissions: 5
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Baylor Genetics | RCV000191143 | SCV000245552 | likely pathogenic | Pontocerebellar hypoplasia type 1A | 2014-01-27 | criteria provided, single submitter | clinical testing | Likely pathogenicity based on finding it once in our laboratory in trans with another missense variant [R321C] in a 33-year-old male with clinical diagnoses of distal spinal muscular atrophy and motor neuropathy. |
Gene |
RCV000523462 | SCV000618460 | pathogenic | not provided | 2023-08-03 | criteria provided, single submitter | clinical testing | Published functional studies demonstrate reduced kinase activity (Martin-Doncel et al., 2019); In silico analysis supports that this missense variant has a deleterious effect on protein structure/function; Missense variants in this gene are often considered pathogenic (HGMD); Not observed at significant frequency in large population cohorts (gnomAD); This variant is associated with the following publications: (PMID: 26633545, 35641352, 31090908, 26583493, 27281532, 37257665, 34169149, 31589614, RodriguezHrnandez2023[abstract], 31527692) |
Fulgent Genetics, |
RCV000191143 | SCV000893357 | likely pathogenic | Pontocerebellar hypoplasia type 1A | 2018-10-31 | criteria provided, single submitter | clinical testing | |
Labcorp Genetics |
RCV000191143 | SCV001588881 | pathogenic | Pontocerebellar hypoplasia type 1A | 2024-02-16 | criteria provided, single submitter | clinical testing | This sequence change replaces histidine, which is basic and polar, with arginine, which is basic and polar, at codon 119 of the VRK1 protein (p.His119Arg). This variant is present in population databases (rs371295780, gnomAD 0.01%). This missense change has been observed in individual(s) with adult onset distal spinal muscular atrophy (PMID: 26583493, 27281532). In at least one individual the data is consistent with being in trans (on the opposite chromosome) from a pathogenic variant. It has also been observed to segregate with disease in related individuals. ClinVar contains an entry for this variant (Variation ID: 209204). An algorithm developed to predict the effect of missense changes on protein structure and function (PolyPhen-2) suggests that this variant is likely to be disruptive. Experimental studies have shown that this missense change affects VRK1 function (PMID: 31527692). For these reasons, this variant has been classified as Pathogenic. |
Fulgent Genetics, |
RCV005008123 | SCV005630511 | likely pathogenic | Pontocerebellar hypoplasia type 1A; Neuronopathy, distal hereditary motor, autosomal recessive 10 | 2024-02-22 | criteria provided, single submitter | clinical testing |