Correction of human phospholamban R14del mutation associated with cardiomyopathy using targeted nucleases and combination therapy

A number of genetic mutations is associated with cardiomyopathies. A mutation in the coding region of the phospholamban (PLN) gene (R14del) is identified in families with hereditary heart failure. Heterozygous patients exhibit left ventricular dilation and ventricular arrhythmias. Here we generate i...

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Detalles Bibliográficos
Autores principales: Karakikes, Ioannis, Stillitano, Francesca, Nonnenmacher, Mathieu, Tzimas, Christos, Sanoudou, Despina, Termglinchan, Vittavat, Kong, Chi-Wing, Rushing, Stephanie, Hansen, Jens, Ceholski, Delaine, Kolokathis, Fotis, Kremastinos, Dimitrios, Katoulis, Alexandros, Ren, Lihuan, Cohen, Ninette, Gho, Johannes M.I.H., Tsiapras, Dimitrios, Vink, Aryan, Wu, Joseph C., Asselbergs, Folkert W., Li, Ronald A., Hulot, Jean-Sebastien, Kranias, Evangelia G., Hajjar, Roger J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Pub. Group 2015
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4421839/
https://www.ncbi.nlm.nih.gov/pubmed/25923014
http://dx.doi.org/10.1038/ncomms7955

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