Total submissions: 4
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Mayo Clinic Laboratories, |
RCV001507456 | SCV001713035 | likely pathogenic | not provided | 2020-02-13 | criteria provided, single submitter | clinical testing | PS3, PM2, PM5 |
Revvity Omics, |
RCV001507456 | SCV003808644 | uncertain significance | not provided | 2020-06-04 | criteria provided, single submitter | clinical testing | |
Women's Health and Genetics/Laboratory Corporation of America, |
RCV004587170 | SCV005077235 | uncertain significance | not specified | 2024-04-18 | criteria provided, single submitter | clinical testing | Variant summary: GBA1 c.593C>T (p.Pro198Leu), also reported as "P159L", results in a non-conservative amino acid change located in the Glycosyl hydrolase family 30, TIM-barrel domain (IPR033453) of the encoded protein sequence. Five of five in-silico tools predict a damaging effect of the variant on protein function. The variant allele was found at a frequency of 2e-06 in 983702 control chromosomes. c.593C>T has been reported as homozygous in the literature in at least one individual affected with type 1 Gaucher Disease (Demina_1998, Petrides_1998). It has also been reported in the heterozygous state in at least one individual with Parkinson's disease (Peterschmitt_2022). These data indicate that the variant may be associated with disease. At least one publication reports that patient cells had ~39% beta-glucocerebrosidase activity vs. control cell minimums (Petrides_1998). The following publications have been ascertained in the context of this evaluation (PMID: 9554454, 34897099, 9723584, 24022302, 12204005). ClinVar contains an entry for this variant (Variation ID: 1162851). Based on the evidence outlined above, the variant was classified as VUS-possibly pathogenic. |
Gene |
RCV001507456 | SCV005324980 | likely pathogenic | not provided | 2024-02-22 | criteria provided, single submitter | clinical testing | In vitro assays support that this variant results in impaired beta-glucocerebrosidase binding with the lysosomal integral membrane protein type-2 (LIMP-2) resulting in defective intercellular targeting to lysosomes. (PMID: 27001828); 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; Also known as p.(P159L); This variant is associated with the following publications: (PMID: 9516376, 9554454, 27001828) |