Total submissions: 5
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
NIHR Bioresource Rare Diseases, |
RCV000018151 | SCV001441155 | likely pathogenic | Telangiectasia, hereditary hemorrhagic, type 1 | 2018-01-01 | criteria provided, single submitter | research | PS3+PM2+PP4 |
Labcorp Genetics |
RCV001851903 | SCV002277734 | likely pathogenic | Hereditary hemorrhagic telangiectasia | 2021-09-24 | criteria provided, single submitter | clinical testing | Nucleotide substitutions within the consensus splice site are a relatively common cause of aberrant splicing (PMID: 17576681, 9536098). Studies have shown that this variant results in skipping of exon 3, but is expected to preserve the integrity of the reading-frame (PMID: 9245986). This sequence change falls in intron 3 of the ENG gene. It does not directly change the encoded amino acid sequence of the ENG protein. RNA analysis indicates that this variant induces altered splicing and likely results in a shortened protein product. This variant is not present in population databases (ExAC no frequency). This variant has been observed in individuals with clinical features of hereditary hemorrhagic telangiectasia (PMID: 9245986, 32573726). ClinVar contains an entry for this variant (Variation ID: 16671). 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. |
Ambry Genetics | RCV004018638 | SCV005037529 | likely pathogenic | Cardiovascular phenotype | 2017-02-20 | criteria provided, single submitter | clinical testing | The c.360+4A>G intronic variant results from an A to G substitution 4 nucleotides after coding exon 3 in the ENG gene. This variant was identified in an individual with hereditary hemorrhagic telangiectasia (HHT) and was reported to result in a donor splice site disruption; mRNA analysis showed exon 3 skipping (Shovlin CL et al. Am. J. Hum. Genet., 1997 Jul;61:68-79). In our internal cohort, this variant was identified in an individual with telangiectasias, epistaxis, and arteriovenous malformations. This variant was not reported in the ExAC database, with coverage at this position. This nucleotide position is highly conserved on limited sequence alignment. Using the BDGP and ESEfinder splice site prediction tools, this alteration is predicted to weaken the efficiency of the native splice donor site; however, direct evidence is unavailable. Based on the majority of available evidence to date, this variant is likely to be pathogenic. |
Gene |
RCV004700247 | SCV005201597 | likely pathogenic | not provided | 2023-11-17 | criteria provided, single submitter | clinical testing | Not observed at significant frequency in large population cohorts (gnomAD); A published functional study showed a splice defect resulting in a truncated protein (PMID: 9245986); In silico analysis supports a deleterious effect on splicing; This variant is associated with the following publications: (PMID: 25525159, 32573726, 9245986) |
OMIM | RCV000018151 | SCV000038430 | pathogenic | Telangiectasia, hereditary hemorrhagic, type 1 | 1997-07-01 | no assertion criteria provided | literature only |