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1) The R453C-betaMHC pathological change was introduced in three independent healthy hPSC lines using CRISPR/Cas9. 2) The gene edited hPSC lines were differentiated to generate isogenic sets of hPSC-cardiomyocytes. 3) Phenotyping of hPSC-CMs in terms of a) molecular, b) functional and c) transcriptomics analyses has validated the human HCM model generated, leading to new mechanistic and pharmacological understanding of the disease.
