Total submissions: 3
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Al Jalila Children’s Genomics Center, |
RCV001733793 | SCV001984726 | likely pathogenic | not specified | 2020-09-13 | criteria provided, single submitter | clinical testing | |
MGZ Medical Genetics Center | RCV002290745 | SCV002581088 | likely pathogenic | Mucopolysaccharidosis, MPS-IV-B | 2022-08-11 | criteria provided, single submitter | clinical testing | |
Women's Health and Genetics/Laboratory Corporation of America, |
RCV002290745 | SCV005076090 | pathogenic | Mucopolysaccharidosis, MPS-IV-B | 2024-04-16 | criteria provided, single submitter | clinical testing | Variant summary: GLB1 c.998A>G (p.Tyr333Cys) results in a non-conservative amino acid change located in the catalytic domain (IPR031330), affecting a residue which is part of the substrate binding pocket (PMID: 22128166) 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 4e-06 in 249536 control chromosomes (gnomAD). c.998A>G has been reported in the literature in 2 homozygous siblings, affected with Mucopolysaccharidosis Type IVB (Morquio Syndrome B) (Mayer_2009, Giugliani_1987). One of these publications also reported experimental evidence evaluating an impact on protein function and demonstrated deficient beta-galactosidase activities in fibroblasts derived from these patients (Giugliani_1987). These data indicate that the variant is likely to be associated with disease. The following publications have been ascertained in the context of this evaluation (PMID: 19091613, 3121219 , 32071837 ). ClinVar contains an entry for this variant (Variation ID: 1301844). Based on the evidence outlined above, the variant was classified as pathogenic. |