Total submissions: 2
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Labcorp Genetics |
RCV001385168 | SCV001584931 | pathogenic | Deficiency of acetyl-CoA acetyltransferase | 2024-01-16 | criteria provided, single submitter | clinical testing | This sequence change affects a donor splice site in intron 11 of the ACAT1 gene. While this variant is not anticipated to result in nonsense mediated decay, it likely alters RNA splicing and results in a disrupted protein product. This variant is present in population databases (no rsID available, gnomAD 0.002%). Disruption of this splice site has been observed in individual(s) with beta-ketothiolase deficiency (Invitae). In at least one individual the data is consistent with being in trans (on the opposite chromosome) from a pathogenic variant. ClinVar contains an entry for this variant (Variation ID: 1072442). 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. This variant disrupts a region of the ACAT1 protein in which other variant(s) (p.Gly418Asp) have been observed in individuals with ACAT1-related conditions (PMID: 28689740). This suggests that this is a clinically significant region of the protein, and that variants that disrupt it are likely to be disease-causing. For these reasons, this variant has been classified as Pathogenic. |
Baylor Genetics | RCV001385168 | SCV005059085 | likely pathogenic | Deficiency of acetyl-CoA acetyltransferase | 2023-12-07 | criteria provided, single submitter | clinical testing |