Why the study?
Current methods for human iPSC-based disease modeling and drug testing are time-consuming and lack an isogenic control.
Does genome editing of iPSCs to introduce dominant negative mutations of KCNQ1 and KCNH2 recapitulate the long QT phenotype compared to unedited controls?
Does genome editing of iPSCs to introduce dominant negative mutations of KCNQ1 and KCNH2 recapitulate the long QT phenotype compared to unedited controls?
Genome editing of iPSCs to overexpress dominant negative gene mutants rapidly generates isogenic disease models of long QT syndrome suitable for drug testing.
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** Enables precise isogenic iPSC models for LQTS drug screening; extends research tools for cardiovascular investigators but leaves open clinical translation.
Wang et al. (2014) studied this question.
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