Total submissions: 1
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Labcorp Genetics |
RCV002002423 | SCV002233881 | pathogenic | Glycogen storage disease, type VII | 2021-04-10 | criteria provided, single submitter | clinical testing | Disruption of this splice site has been observed in individual(s) with glycogen storage disease (PMID: 8444874, 8037209). This variant is not present in population databases (ExAC no frequency). This sequence change affects a donor splice site in intron 4 of the PFKM gene. RNA analysis indicates that this variant induces altered splicing and likely results in a shortened protein product. Studies have shown that disruption of this splice site is associated with skipping of exon 4 (also known as exon 5) but is expected to preserve the integrity of the reading frame (PMID:8444874). For these reasons, this variant has been classified as Pathogenic. |