Total submissions: 1
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Laboratory for Molecular Medicine, |
RCV000190636 | SCV000245679 | pathogenic | Johanson-Blizzard syndrome | 2014-09-30 | criteria provided, single submitter | clinical testing | The Cys1369X variant in UBR1 has not been previously reported in individuals with Johanson-Blizzard syndrome (JBS) or in large population studies. This nonsense variant leads to a premature termination codon at position 1369 which is predicted to lead to a truncated or absent protein. Complete loss of UBR1 function is an established disease mechanism in JBS (http://omim.org/entry/605981) .In summary, this variant meets our criteria to be classified as pathogenic for JBS in an autosomal recessive manner (http://personalizedmedicine.partners.org/Laboratory-For-Molecular-Medicine/). |