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CRISPR/Cas9 editing in human pluripotent stem cell-cardiomyocytes highlights arrhythmias, hypocontractility, and energy depletion as potential therapeutic targets for hypertrophic cardiomyopathy

AIMS: Sarcomeric gene mutations frequently underlie hypertrophic cardiomyopathy (HCM), a prevalent and complex condition leading to left ventricle thickening and heart dysfunction. We evaluated isogenic genome-edited human pluripotent stem cell-cardiomyocytes (hPSC-CM) for their validity to model, a...

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Detalles Bibliográficos
Autores principales: Mosqueira, Diogo, Mannhardt, Ingra, Bhagwan, Jamie R, Lis-Slimak, Katarzyna, Katili, Puspita, Scott, Elizabeth, Hassan, Mustafa, Prondzynski, Maksymilian, Harmer, Stephen C, Tinker, Andrew, Smith, James G W, Carrier, Lucie, Williams, Philip M, Gaffney, Daniel, Eschenhagen, Thomas, Hansen, Arne, Denning, Chris
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6234851/
https://www.ncbi.nlm.nih.gov/pubmed/29741611
http://dx.doi.org/10.1093/eurheartj/ehy249