Total submissions: 2
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Labcorp Genetics |
RCV002848255 | SCV003235754 | pathogenic | Mucopolysaccharidosis, MPS-III-C; Retinitis pigmentosa 73 | 2023-12-05 | criteria provided, single submitter | clinical testing | This sequence change affects an acceptor splice site in intron 4 of the HGSNAT gene. It is expected to disrupt RNA splicing. Variants that disrupt the donor or acceptor splice site typically lead to a loss of protein function (PMID: 16199547), and loss-of-function variants in HGSNAT are known to be pathogenic (PMID: 17033958, 19479962). This variant is not present in population databases (gnomAD no frequency). Disruption of this splice site has been observed in individual(s) with Mucopolysaccharidosis type III (PMID: 31228227). ClinVar contains an entry for this variant (Variation ID: 2029865). Studies have shown that disruption of this splice site is associated with altered splicing resulting in multiple RNA products (PMID: 31228227). For these reasons, this variant has been classified as Pathogenic. |
Fulgent Genetics, |
RCV002848255 | SCV005675482 | likely pathogenic | Mucopolysaccharidosis, MPS-III-C; Retinitis pigmentosa 73 | 2024-04-10 | criteria provided, single submitter | clinical testing |