Total submissions: 1
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Labcorp Genetics |
RCV001928878 | SCV002201067 | pathogenic | Bifunctional peroxisomal enzyme deficiency; Perrault syndrome | 2021-04-21 | criteria provided, single submitter | clinical testing | This sequence change creates a premature translational stop signal (p.Thr494Argfs*31) in the HSD17B4 gene. It is expected to result in an absent or disrupted protein product. Loss-of-function variants in HSD17B4 are known to be pathogenic (PMID: 11810648, 16385454). This variant is present in population databases (rs775060894, ExAC 0.001%). For these reasons, this variant has been classified as Pathogenic. Algorithms developed to predict the effect of sequence changes on RNA splicing suggest that this variant may create or strengthen a splice site, but this prediction has not been confirmed by published transcriptional studies. This variant has not been reported in the literature in individuals with HSD17B4-related conditions. |