Total submissions: 4
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Gene |
RCV000498035 | SCV000589624 | pathogenic | not provided | 2018-08-17 | criteria provided, single submitter | clinical testing | The c.254+1 G>A variant has been reported in a patient with mitochondrial trifunctional protein deficiency who harbored a missense variant on the opposite HADHB allele (in trans) (Terrone et al. 2014). The c.254+1 G>A variant destroys the canonical splice donor site in intron 5. Sequencing of the reported patient's mRNA revealed both a wild-type transcript and a transcript lacking exon 5 consistent with c.254+1 G>A causing abnormal gene splicing (Terrone et al. 2014). The c.254+1 G>A variant is not observed in large population cohorts (Lek et al., 2016; 1000 Genomes Consortium et al., 2015; Exome Variant Server). In summary, we interpret this variant as pathogenic. |
Fulgent Genetics, |
RCV000763080 | SCV000893603 | pathogenic | Mitochondrial trifunctional protein deficiency | 2018-10-31 | criteria provided, single submitter | clinical testing | |
Baylor Genetics | RCV004568626 | SCV004191812 | pathogenic | Mitochondrial trifunctional protein deficiency 1 | 2024-03-27 | criteria provided, single submitter | clinical testing | |
Labcorp Genetics |
RCV000763080 | SCV004292065 | likely pathogenic | Mitochondrial trifunctional protein deficiency | 2023-05-20 | criteria provided, single submitter | clinical testing | 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. Studies have shown that disruption of this splice site results in skipping of exon 5, but is expected to preserve the integrity of the reading-frame (PMID: 24379101). ClinVar contains an entry for this variant (Variation ID: 431987). Disruption of this splice site has been observed in individual(s) with HADHB-related conditions (PMID: 24379101). This variant is present in population databases (rs776172237, gnomAD 0.008%). This sequence change affects a donor splice site in intron 5 of the HADHB gene. RNA analysis indicates that disruption of this splice site induces altered splicing and likely results in a shortened protein product. |