Article

Variable expression and silencing of CRISPR-Cas9 targeted transgenes identifies the AAVS1 locus as not an entirely safe harbour

2019-11-12

Abstract excerpt

<ns4:p> <ns4:bold>Background:</ns4:bold> Diseases such as hypertrophic cardiomyopathy (HCM) can lead to severe outcomes including sudden death. The generation of human induced pluripotent stem cell (hiPSC) reporter lines can be useful for disease modelling and drug screening by providing physiologically relevant <ns4:italic>in vitro</ns4:italic> models of disease. The <ns4:italic>AAVS1</ns4:italic> locus is cited...

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Literature Corpus work: 24818236-cc14-56ec-bb17-1fdcaf7a289e
DOI: 10.12688/f1000research.19894.1

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