Total submissions: 4
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Institute of Human Genetics, |
RCV001260592 | SCV001397587 | likely pathogenic | Adrenoleukodystrophy | 2020-07-13 | criteria provided, single submitter | clinical testing | The variant c.851C>T (p.(Ser284Leu)) in exon 1 of the ABCD1-gene is not found in known databases (ExAC or gnomAD), it affects a highly conserved nucleotide, a highly conserved amino acid and there is a large physicochemical difference between Ser and Leu. This variant is located in a mutational hotspot and within a protein domain and has a pathogenic computational verdict based on 8 pathogenic predictions from DANN, DEOGEN2, FATHMM-MKL, M-CAP, MVP, MutationTaster, REVEL and PolyPhen-2 vs 2 benign predictions from MutationAssessor and SIFT. Our male patient, carrying this mutation showed increased plasma levels of very long chain fatty acids (VLCFAs) and a phenotype typical for the adult form of X-linked adrenoleukodystrophy. Thus we consider this ABCD1-variant to be likely pathogenic. ACMG criteria used for classification: PM1, PM2, PP2, PP3, PP4. |
MGZ Medical Genetics Center | RCV001260592 | SCV002580814 | likely pathogenic | Adrenoleukodystrophy | 2022-02-15 | criteria provided, single submitter | clinical testing | |
Invitae | RCV001260592 | SCV003471423 | pathogenic | Adrenoleukodystrophy | 2023-04-03 | criteria provided, single submitter | clinical testing | Advanced modeling of protein sequence and biophysical properties (such as structural, functional, and spatial information, amino acid conservation, physicochemical variation, residue mobility, and thermodynamic stability) performed at Invitae indicates that this missense variant is expected to disrupt ABCD1 protein function. For these reasons, this variant has been classified as Pathogenic. ClinVar contains an entry for this variant (Variation ID: 981244). This missense change has been observed in individual(s) with adrenoleukodystrophy (Priestley JR et al. 2022. Int. J. Neonatal Screen. 8(2); ClinVar; Invitae). This variant is not present in population databases (gnomAD no frequency). This sequence change replaces serine, which is neutral and polar, with leucine, which is neutral and non-polar, at codon 284 of the ABCD1 protein (p.Ser284Leu). |
Revvity Omics, |
RCV003135895 | SCV003824431 | uncertain significance | not provided | 2022-02-17 | criteria provided, single submitter | clinical testing |