Total submissions: 2
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Clin |
RCV004821979 | SCV005442790 | likely pathogenic | Phenylketonuria | 2024-09-06 | reviewed by expert panel | curation | The NM_000277.1(PAH):c.922C>G (p.Leu308Val) variant is a missense variant in exon 9/13 of PAH. The variant has been found to reduce PAH enzymatic activity to 15-19% versus wild type PAH enzyme in transfected COS cells (PMID: 18590700). The variant has been previously reported in confirmed trans with the c.1315+1G>A variant (Pathogenic per ClinGen PAH VCEP) in a patient with mild PKU (plasma Phe 600-900 umol/L); BH4 deficiency does not appear to have been excluded (PMID: 26542770). The variant is absent from ethnically diverse control databases, including gnomAD/ExAC, 1000 Genomes, and ESP. The variant is predicted damaging by multiple in-silico missense predictors, including REVEL (REVEL score 0.892). Classification: Likely Pathogenic Supporting Criteria: PS3_supporting; PM2_supporting; PM3; PP4; PP3_moderate |
De |
RCV000089158 | SCV000119772 | not provided | not provided | no assertion provided | not provided |