Total submissions: 2
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Labcorp Genetics |
RCV000014303 | SCV002247266 | pathogenic | Sphingolipid activator protein 1 deficiency | 2021-05-18 | criteria provided, single submitter | clinical testing | Studies have shown that disruption of this splice site is associated with partial and complete skipping of exon 6 but is expected to preserve the integrity of the reading frame (PMID: 8554069). For these reasons, this variant has been classified as Pathogenic. Experimental studies have shown that this variant affects PSAP protein function (PMID: 8554069). Disruption of this splice site has been observed in individual(s) with saposin B deficiency and metachromatic leukodystrophy (PMID: 19267410, 26462614). ClinVar contains an entry for this variant (Variation ID: 13374). This variant is not present in population databases (ExAC no frequency). This sequence change affects an acceptor splice site in intron 5 of the PSAP gene. RNA analysis indicates that this variant induces altered splicing and likely results in a shortened protein product. |
OMIM | RCV000014303 | SCV000034552 | pathogenic | Sphingolipid activator protein 1 deficiency | 2009-02-15 | no assertion criteria provided | literature only |