Total submissions: 1
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Clin |
RCV004823288 | SCV005442791 | likely pathogenic | Phenylketonuria | 2024-09-06 | reviewed by expert panel | curation | The c.686A>G (p.Asp229Gly) variant in PAH has been reported in 1 individual with PAH deficiency, detected with pathogenic variant p.R408W (PMID: 23357515). This variant is absent in population databases. Computational evidence supports a deleterious effect (REVEL=0.922). Functional studies show the p.Asp229Gly mutant had 0% residual activity as compared to WT PAH activity and decreased protein levels (PMID: 31208052). In summary, this variant meets criteria to be classified as likely pathogenic for PAH. PAH-specific ACMG/AMP criteria applied: PS3_supporting, PP4, PM2_supporting, PM3_supporting, PP3_moderate. |