Total submissions: 1
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Ambry Genetics | RCV002405831 | SCV002705944 | pathogenic | Diamond-Blackfan anemia | 2016-09-09 | criteria provided, single submitter | clinical testing | The c.158-2A>C intronic pathogenic mutation results from an A to C substitution two nucleotides upstream from coding exon 3 in the RPL11 gene. This mutation was reported in a patient with Diamond-Blackfan anemia (DBA) with growth retardation but no malformations; he was reported to be responsive to steroids and had no family history of DBA (Boria I et al. Hum. Mutat., 2010 Dec;31:1269-79). In addition to the clinical data presented in the literature, alterations that disrupt the canonical splice site are expected to cause aberrant splicing, resulting in an abnormal protein or a transcript that is subject to nonsense-mediated mRNA decay. As such, this alteration is classified as pathogenic. |