Total submissions: 2
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Clin |
RCV002274266 | SCV002558820 | pathogenic | Monogenic diabetes | 2022-07-01 | reviewed by expert panel | curation | The c.404del variant in the HNF1 Homeobox A gene, HMF1A, causes a frameshift in the protein at codon 135 (NM_000545.8), adding 20 novel amino acids before encountering a stop codon (p.(Asp135ValfsTer20)). This variant, located in biologically-relevant exon 2 of 10, is predicted to lead 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), and was identified in 9 unrelated individuals with non- autoimmune and non-absolute/near-absolute insulin-deficient diabetes (PS4; PMID 30269055, internal lab contributors). At least one of these individuals has a clinical history highly specific for HNF1A-MODY (MODY probability calculator result >50%, negative genetic testing for HNF4A, and sensitive to sulfonylureas) (PP4_Moderate; PMID 30269055, internal lab contributor). This variant also segregated with diabetes, with at least 11 informative meioses in six families with MODY (PP1_Strong; internal lab contributor). In summary, the c.404del variant meets the criteria to be classified as pathogenic for monogenic diabetes. ACMG/AMP criteria applied, as specified by the ClinGen MDEP (specification version 1.1, approved 9/30/21): PVS1, PP1_Strong, PS4, PP4_Moderate, PM2_Supporting. |
Genetics and Molecular Pathology, |
RCV002466752 | SCV002761915 | likely pathogenic | Maturity-onset diabetes of the young type 3 | 2021-10-07 | criteria provided, single submitter | clinical testing |