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

Autor: Daniel J. Gaffney, James G W Smith, Ingra Mannhardt, Lucie Carrier, Philip M. Williams, Diogo Mosqueira, Stephen C Harmer, Arne Hansen, Chris Denning, Katarzyna Lis-Slimak, Thomas Eschenhagen, Maksymilian Prondzynski, Puspita A Katili, Andrew Tinker, Jamie R. Bhagwan, Mustafa Hassan, Elizabeth Scott

Publikováno v: Mosqueira, D, Mannhardt, I, Bhagwan, J R, Lis-Slimak, K, Katili, P, Scott, E, Hassan, M, Prondzynski, M, Harmer, S C, Tinker, A, Smith, J G W, Carrier, L, Williams, P M, Gaffney, D, Eschenhagen, T, Hansen, A & Denning, C 2018, ' CRISPR/Cas9 editing in human pluripotent stem cell-cardiomyocytes highlights arrhythmias, hypocontractility, and energy depletion as potential therapeutic targets for hypertrophic cardiomyopathy ', European Heart Journal, vol. 39, no. 43, pp. 3879-3892 . https://doi.org/10.1093/eurheartj/ehy249
European Heart Journal

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-cardi

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https://ueaeprints.uea.ac.uk/id/eprint/69544/