Total submissions: 4
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Clin |
RCV001332014 | SCV003852684 | uncertain significance | Arginine:glycine amidinotransferase deficiency | 2023-01-24 | reviewed by expert panel | curation | The NM_001482.3:c.1022C>T variant in GATM is a missense variant predicted to cause substitution of proline by leucine at amino acid 341 (p.Pro341Leu). This variant is absent from gnomAD v2.1.1 (PM2_Supporting). The computational predictor REVEL gives a score of 0.476 which is neither above nor below the thresholds predicting a damaging (>0.75) or benign (<0.15) impact on AGAT function. There is a ClinVar entry for this variant (Variation ID: 917496). To our knowledge, this variant has not been reported in an individual with phenotypic features of AGAT deficiency. It has been reported as a single heterozygous variant segregating in a family autosomal dominant renal Fanconi syndrome and kidney failure (PMID: 29654216). However, classification with respect to this alternative disorder is outside the scope of this curation. There is a ClinVar entry for this variant (Variation ID: 917496). In summary, this variant meets the criteria to be classified as a variant of uncertain significance for AGAT deficiency based on the ACMG/AMP criteria applied, as specified by the ClinGen Cerebral Creatine Deficiency Syndromes Variant Curation Expert Panel (Specifications Version 1.1.0): PM2_Supporting. (Classification approved by the ClinGen CCDS VCEP on January 24, 2023). |
Baylor Genetics | RCV001332014 | SCV001524197 | uncertain significance | Arginine:glycine amidinotransferase deficiency | 2019-02-14 | criteria provided, single submitter | clinical testing | This variant was determined to be of uncertain significance according to ACMG Guidelines, 2015 [PMID:25741868]. This variant has been previously reported as disease causing [PMID 29654216] |
Fulgent Genetics, |
RCV002483940 | SCV002791731 | uncertain significance | Arginine:glycine amidinotransferase deficiency; Fanconi renotubular syndrome 1 | 2022-04-30 | criteria provided, single submitter | clinical testing | |
OMIM | RCV001174512 | SCV001337651 | pathogenic | Fanconi renotubular syndrome 1 | 2020-06-11 | no assertion criteria provided | literature only |