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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Detalles Bibliográficos
Autores principales: Bednarski, Christien, Tomczak, Katja, vom Hövel, Beate, Weber, Wolf-Michael, Cathomen, Toni
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2016
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4985144/
https://www.ncbi.nlm.nih.gov/pubmed/27526025
http://dx.doi.org/10.1371/journal.pone.0161072

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