Total submissions: 2
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Labcorp Genetics |
RCV001977210 | SCV002260737 | likely pathogenic | Deficiency of hydroxymethylglutaryl-CoA lyase | 2023-04-26 | criteria provided, single submitter | clinical testing | This variant is present in population databases (rs775218067, gnomAD 0.006%). Disruption of this splice site has been observed in individual(s) with 3-hydroxy-3-methylglutaryl-CoA lyase deficiency (PMID: 11129331, 19036343). This variant is also known as IVS3 + 1G>A. ClinVar contains an entry for this variant (Variation ID: 1472739). Algorithms developed to predict the effect of variants on protein structure and function are not available or were not evaluated for this variant. Experimental studies have shown that disruption of this splice site affects HMGCL function (PMID: 11129331). Studies have shown that disruption of this splice site results in skipping of exon 3, but is expected to preserve the integrity of the reading-frame (PMID: 19036343). In summary, the currently available evidence indicates that the variant is pathogenic, but additional data are needed to prove that conclusively. Therefore, this variant has been classified as Likely Pathogenic. This sequence change affects a donor splice site in intron 3 of the HMGCL gene. RNA analysis indicates that disruption of this splice site induces altered splicing and likely results in a shortened protein product. |
Baylor Genetics | RCV001977210 | SCV004199892 | pathogenic | Deficiency of hydroxymethylglutaryl-CoA lyase | 2023-08-07 | criteria provided, single submitter | clinical testing |