Article
Targeted Integration of a Super-Exon into the CFTR Locus Leads to Functional Correction of a Cystic Fibrosis Cell Line Model.
PloS one - 1 Jan 2016
Bednarski Christien, Tomczak Katja, Vom Hövel Beate, Weber Wolf-Michael, Cathomen Toni
Abstract excerpt
In vitro disease models have enabled insights into the pathophysiology of human disease as well as the functional evaluation of new therapies, such as novel genome engineering strategies. In the context of cystic fibrosis (CF), various cellular disease models have been established in recent years, including organoids based on induced pluripotent stem cell technology that allowed for functional readouts of CFTR...
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