Total submissions: 1
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Broad Center for Mendelian Genomics, |
RCV003226056 | SCV003922150 | likely pathogenic | Insulin-dependent diabetes mellitus secretory diarrhea syndrome | 2023-05-02 | criteria provided, single submitter | curation | The hemizygous c.542+2T>G variant in FOXP3 was identified by our study in one individual with X-linked immunodysregulation, polyendocrinopathy, and enteropathy (IPEX). The c.542+2T>G variant in FOXP3 has not been previously reported in individuals with IPEX. This variant was absent from large population studies. This variant is located in the 5' splice region. Computational tools predict a splicing impact, though this information is not predictive enough to determine pathogenicity. Loss of function of the FOXP3 gene is an established disease mechanism in X-linked recessive IPEX. In summary, although additional studies are required to fully establish its clinical significance, this variant is likely pathogenic for X-linked IPEX. ACMG/AMP Criteria applied: PVS1, PM2_Supporting (Richards 2015). |