Total submissions: 2
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Clin |
RCV004574960 | SCV005050169 | pathogenic | Monogenic diabetes | 2024-05-09 | reviewed by expert panel | curation | The c.583-1G>A variant in the hepatocyte nuclear factor4-alpha gene, HNF4A, is predicted to remove a canonical splice acceptor site in intron 5 of NM_175914.5. This variant is predicted to cause skipping of biologically-relevant exon 6 of 10, resulting in a frameshift, leading to nonsense mediated decay in a gene in which loss-of-function is an established disease mechanism (PVS1; PMID: 23348805). This variant is absent in gnomAD v2.1.1 (PM2_Supporting). It was identified in three unrelated individuals with non-autoimmune and non-absolute/near-absolute insulin-deficient diabetes; however, PS4_Moderate cannot be applied because this number is below the ClinGen MDEP threshold (internal lab contributors). One of these individuals has a clinical history highly specific for HNF4A-MODY (MODY probability calculator result >50%, negative genetic testing for HNF1A) (PP4; internal lab contributor). In summary, c.583-1G>A meets the criteria to be classified as pathogenic for monogenic diabetes. ACMG/AMP criteria applied, as specified by the ClinGen MDEP (specification version 2.0.0, approved 10/11/2023): PVS1, PP4, PM2_Supporting. |
Juno Genomics, |
RCV004796870 | SCV005416962 | pathogenic | Maturity-onset diabetes of the young type 1; Type 2 diabetes mellitus; Fanconi renotubular syndrome 4 with maturity-onset diabetes of the young | criteria provided, single submitter | clinical testing | PM2_Supporting+PVS1+PP4 |