Total submissions: 3
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Tartaglia Lab, |
RCV000754393 | SCV000786644 | likely pathogenic | Neonatal pseudo-hydrocephalic progeroid syndrome | 2018-05-01 | criteria provided, single submitter | research | |
Neurometabolic Diseases Laboratory, |
RCV003387902 | SCV003920805 | likely pathogenic | POLR3A-related disorder | 2023-04-27 | criteria provided, single submitter | research | |
Broad Center for Mendelian Genomics, |
RCV003488782 | SCV004232693 | uncertain significance | Leukodystrophy | 2024-01-24 | criteria provided, single submitter | curation | The p.Asp1292Asn variant in POLR3A has been reported in 1 individual with POLR3A-related disorders but has been identified in 0.005% (1/21636) of European (Finnish) chromosomes by the Genome Aggregation Database (gnomAD, http://gnomad.broadinstitute.org; dbSNP ID: rs757209071). Although this variant has been seen in the general population in a heterozygous state, its frequency is low enough to be consistent with a recessive carrier frequency. This variant has also been reported in ClinVar (Variation ID#: 549571) and has been interpreted as likely pathogenic by Tartaglia Lab (Genetics and Rare Diseases Research Division, Bambino Gesu' Children's Hospital). The p.Asp1292Asn variant is located in a region of POLR3A that is essential to protein folding and stability, suggesting that this variant is in a hot spot/functional domain and slightly supports pathogenicity (PMID: 30323018). In summary, while there is some suspicion for a pathogenic role, the clinical significance of this variant is uncertain. ACMG/AMP Criteria applied: PP3, PM1_supporting, PM2_supporting (Richards 2015). |