Total submissions: 1
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Laboratory for Molecular Medicine, |
RCV000825540 | SCV000966856 | likely pathogenic | Hyperphosphatasia-intellectual disability syndrome | 2018-09-21 | criteria provided, single submitter | clinical testing | The p.Pro496SerfsX53 variant in PIGO has not been previously reported in individ uals with hyperphosphatasia-intellectual disability syndrome, also known as Mabr y syndrome, and was absent from large population studies. This variant is predic ted to cause a frameshift, which alters the protein?s amino acid sequence beginn ing at position 496 and leads to a premature termination codon 53 amino acids do wnstream. This alteration is then predicted to lead to a truncated or absent pro tein. Loss of function of the PIGO gene is strongly associated to autosomal rec essive hyperphosphatasia-intellectual disability syndrome. In summary, although additional studies are required to fully establish its clinical significance, th e p.Pro496SerfsX53 variant is likely pathogenic. ACMG/AMP Criteria applied: PVS1 , PM2. |