Total submissions: 3
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Al Jalila Children’s Genomics Center, |
RCV004798717 | SCV005420808 | likely pathogenic | Congenital bile acid synthesis defect | 2024-10-04 | criteria provided, single submitter | research | PS3,PM2,PP3 |
Women's Health and Genetics/Laboratory Corporation of America, |
RCV000005709 | SCV005422519 | pathogenic | Congenital bile acid synthesis defect 2 | 2024-10-09 | criteria provided, single submitter | clinical testing | Variant summary: AKR1D1 c.781C>T (p.Arg261Cys) results in a non-conservative amino acid change in the encoded protein sequence. Four of five in-silico tools predict a damaging effect of the variant on protein function. The variant allele was found at a frequency of 1.2e-05 in 251400 control chromosomes (gnomAD). c.781C>T has been reported in the literature in individuals affected with Congenital bile acid synthesis defect 2 (e.g. Gonzales_2004, Seki_2013). These data indicate that the variant is likely to be associated with disease. At least one publication reports experimental evidence evaluating an impact on protein function, finding that the variant results in sharpy reduced enzymatic activity (Drury_2010). The following publications have been ascertained in the context of this evaluation (PMID: 15030995, 20522910, 23160874). ClinVar contains an entry for this variant (Variation ID: 5378). Based on the evidence outlined above, the variant was classified as pathogenic. |
OMIM | RCV000005709 | SCV000025891 | pathogenic | Congenital bile acid synthesis defect 2 | 2004-04-01 | no assertion criteria provided | literature only |