Total submissions: 3
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Labcorp Genetics |
RCV000023577 | SCV003443372 | likely pathogenic | Aicardi-Goutieres syndrome 5 | 2023-09-25 | criteria provided, single submitter | clinical testing | This sequence change replaces arginine, which is basic and polar, with cysteine, which is neutral and slightly polar, at codon 143 of the SAMHD1 protein (p.Arg143Cys). This variant is present in population databases (rs387906948, gnomAD 0.0009%). This missense change has been observed in individual(s) with Aicardi-Goutieres syndrome (PMID: 19525956). In at least one individual the data is consistent with being in trans (on the opposite chromosome) from a pathogenic variant. ClinVar contains an entry for this variant (Variation ID: 30604). 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 SAMHD1 protein function. Experimental studies have shown that this missense change affects SAMHD1 function (PMID: 26431200, 28229507). Algorithms developed to predict the effect of sequence changes on RNA splicing suggest that this variant may disrupt the consensus splice site. This variant disrupts the p.Arg143 amino acid residue in SAMHD1. Other variant(s) that disrupt this residue have been determined to be pathogenic (PMID: 19525956, 28229507, 29379009, 33683010; Invitae). This suggests that this residue is clinically significant, and that variants that disrupt this residue are likely to be disease-causing. In summary, the currently available evidence indicates that the variant is pathogenic, but additional data are needed to prove that conclusively. Therefore, this variant has been classified as Likely Pathogenic. |
OMIM | RCV000023577 | SCV000044868 | pathogenic | Aicardi-Goutieres syndrome 5 | 2009-07-01 | no assertion criteria provided | literature only | |
Gene |
RCV000023577 | SCV000147930 | not provided | Aicardi-Goutieres syndrome 5 | no assertion provided | literature only |