Total submissions: 2
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Women's Health and Genetics/Laboratory Corporation of America, |
RCV003236250 | SCV003933932 | likely pathogenic | Adrenoleukodystrophy | 2023-05-04 | criteria provided, single submitter | clinical testing | Variant summary: ABCD1 c.220C>T (p.Arg74Trp) results in a non-conservative amino acid change in the encoded protein sequence. Five of five in-silico tools predict a damaging effect of the variant on protein function. The variant was absent in 126597 control chromosomes. c.220C>T has been reported in the literature in individuals affected with Adrenoleukodystrophy (e.g. Engelen_2014, vandeStadt_2021). ABCD1 protein is absent in these patients. At least one publication reports experimental evidence and showed that this variant caused reduced expression of ABCD1 protein (Zhang_2011). The following publications have been ascertained in the context of this evaluation (PMID: 24480483, 14533738, 21476988, 34946879). No clinical diagnostic laboratories have submitted clinical-significance assessments for this variant to ClinVar after 2014. Based on the evidence outlined above, the variant was classified as likely pathogenic. |
Gene |
RCV004593245 | SCV005081036 | likely pathogenic | not provided | 2023-12-08 | criteria provided, single submitter | clinical testing | Reported previously in three female individuals from the same family including one individual with a gait disorder, sensory disturbance, spasticity, weakness, and pathological reflexes; the other two individuals had no symptoms reported (PMID: 24480483); Published functional studies showed a non-significant difference in peroxismal targeting (PMID: 14533738); In silico analysis supports that this missense variant has a deleterious effect on protein structure/function; Not observed at significant frequency in large population cohorts (gnomAD); This variant is associated with the following publications: (PMID: 21476988, 34946879, 24480483, 14533738) |