Total submissions: 2
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Ambry Genetics | RCV002417107 | SCV002724200 | pathogenic | Diamond-Blackfan anemia | 2015-03-25 | criteria provided, single submitter | clinical testing | The p.M1? pathogenic mutation (also known as c.1A>G), located in coding exon 1 of the RPS17 gene, results from an A to G substitution at nucleotide position 1. This alters the methionine residue at the initiation codon. This mutation has been described to occur de novo in an infant with a clinical diagnosis of Diamond Blackfan Anemia (Song MJ, Pediatr Blood Cancer 2010 Apr; 54(4):629-31). In addition to the clinical data presented in the literature, sequence variations that modify the initiation codon (ATG) are expected to result in either loss of translation initiation or N-terminal truncation, this alteration is interpreted as a disease-causing mutation (ACMG Recommendations for Standards for Interpretation and Reporting of Sequence Variations. Revision 2007. Genet Med. 2008;10:294). |
Genetic Services Laboratory, |
RCV003151406 | SCV003839994 | likely pathogenic | not provided | 2022-02-25 | no assertion criteria provided | clinical testing | DNA sequence analysis of the RPS17 gene demonstrated a sequence change, c.1A>G, in exon 1 that impacts the initiator methionine of the RPS17 mRNA. This sequence change may result in an absent or abnormal transcript. This sequence change has been reported in an individual with a diagnosis of Diamond-Blackfan anemia and it was confirmed to be de novo (PMID: 19953637). Additionally, a different sequence change also impacting the initiation codon, c.2T>G, was identified as a de novo variant in a patient with Diamond-Blackfan anemia presenting as macrocytic anemia with increased activity of erythrocyte adenosine deaminase, short stature, facial dysmorphism, and a flat thenar eminence (PMID: 17647292). This sequence change has not been described in population databases such as ExAC and gnomAD. Based on these evidences, this sequence change is likely pathogenic, however functional studies have not been performed to prove this conclusively. |