Total submissions: 1
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Laboratório de Neurologia Aplicada e Experimental, |
RCV002221639 | SCV001977113 | likely pathogenic | Charcot-Marie-Tooth disease type 2 | 2021-07-20 | criteria provided, single submitter | research | The c.1113+2T>A variant in the SLC5A7 gene has not been reported in the literature in individuals with SLC5A7-related conditions. Variants in this gene are responsible for congenital myasthenic syndrome type 20 (AR-CMS20; OMIM: 617143) and dHMN type VIIA (AD; OMIM: 158580). Our lab found it once, in homozygous, in a 1-years-old male with CMT2 phenotype. This variant is in a canonical splice-site and is predicted to affect mRNA splicing, usually leading to a loss of protein function (PMID: 25741868; PMID: 30192042). This variant is not present in population databases (GnomAD and ABraOM), suggesting it is not a common benign variant in these populations. In summary, the c.1113+2T>A variant meets our criteria to be classified as likely pathogenic. |