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Targeted Integration of a Super-Exon into the CFTR Locus Leads to Functional Correction of a Cystic Fibrosis Cell Line Model

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, i...

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Vydáno v:PLoS One
Hlavní autoři: Bednarski, Christien, Tomczak, Katja, vom Hövel, Beate, Weber, Wolf-Michael, Cathomen, Toni
Médium: Artigo
Jazyk:Inglês
Vydáno: Public Library of Science 2016
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On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC4985144/
https://ncbi.nlm.nih.gov/pubmed/27526025
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0161072
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