Calvete Oriol, Martinez Paula, Garcia-Pavia Pablo, Benitez-Buelga Carlos, Paumard-Hernández Beatriz, Fernandez Victoria, Dominguez Fernando, Salas Clara, Romero-Laorden Nuria, Garcia-Donas Jesus, Carrillo Jaime, Perona Rosario, Triviño Juan Carlos, Andrés Raquel, Cano Juana María, Rivera Bárbara, Alonso-Pulpon Luis, Setien Fernando, Esteller Manel, Rodriguez-Perales Sandra, Bougeard Gaelle, Frebourg Tierry, Urioste Miguel, Blasco Maria A, Benítez Javier
Human Genetics Group, Spanish National Cancer Research Center (CNIO), Melchor Fernandez Almagro 3, Madrid 28029, Spain.
Center for Biomedical Network Research on Rare Diseases (CIBERER), Madrid 28029, Spain.
Nat Commun. 2015 Sep 25;6:8383. doi: 10.1038/ncomms9383.
Cardiac angiosarcoma (CAS) is a rare malignant tumour whose genetic basis is unknown. Here we show, by whole-exome sequencing of a TP53-negative Li-Fraumeni-like (LFL) family including CAS cases, that a missense variant (p.R117C) in POT1 (protection of telomeres 1) gene is responsible for CAS. The same gene alteration is found in two other LFL families with CAS, supporting the causal effect of the identified mutation. We extend the analysis to TP53-negative LFL families with no CAS and find the same mutation in a breast AS family. The mutation is recently found once in 121,324 studied alleles in ExAC server but it is not described in any other database or found in 1,520 Spanish controls. In silico structural analysis suggests how the mutation disrupts POT1 structure. Functional and in vitro studies demonstrate that carriers of the mutation show reduced telomere-bound POT1 levels, abnormally long telomeres and increased telomere fragility.
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