Total submissions: 1
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Laboratory for Molecular Medicine, |
RCV000156104 | SCV000205817 | likely pathogenic | Arrhythmogenic right ventricular cardiomyopathy | 2015-04-08 | criteria provided, single submitter | clinical testing | The p.Thr851fsExtX50 variant in PKP2 has not been previously reported in any oth er families with cardiomyopathy or in large population studies. This variant is predicted to cause a frameshift at amino acid 851 which alters the terminal 30 a mino acids and the stop codon such that an additional 50 amino acids are generat ed before introducing a new stop codon. Heterozygous loss of PKP2 function is an established disease mechanism for ARVC; however it is unclear how the p.Thr851f sExtX50 variant would impact the protein function. Of note, 4 other variants hav ing a similar impact to the protein (p.Ser837fsExtX50, p.Leu847fsExtX50, p.Glu85 2fsExtX50, p.Lys859fsExtX50) have been described in >15 individuals with ARVC (G erull 2004, Antoniades 2006, Dalal 2006, Asimaki 2009, Dalal 2009, Fressart 2010 , Den Haan 2009, Watkins 2009, Qiu 2009, Xu 2010, Barahona-Dussault 2010, Tan, 2 010, Cox 2011, Baskin 2013, Alcalde 2014), suggesting that PKP2 protein extensio n variants are disease-causing. In summary, although additional studies are requ ired to fully establish its clinical significance, the p.Thr851fsExtX50 variant is likely pathogenic. |