Functional Repair of CFTR by CRISPR/Cas9 in Intestinal Stem Cell Organoids of Cystic Fibrosis Patients

Single murine and human intestinal stem cells can be expanded in culture over long time periods as genetically and phenotypically stable epithelial organoids. Increased cAMP levels induce rapid swelling of such organoids by opening the cystic fibrosis transmembrane conductor receptor (CFTR). This re...

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Veröffentlicht in:	Cell stem cell 2013-12, Vol.13 (6), p.653-658
Hauptverfasser:	Schwank, Gerald, Koo, Bon-Kyoung, Sasselli, Valentina, Dekkers, Johanna F., Heo, Inha, Demircan, Turan, Sasaki, Nobuo, Boymans, Sander, Cuppen, Edwin, van der Ent, Cornelis K., Nieuwenhuis, Edward E.S., Beekman, Jeffrey M., Clevers, Hans
Format:	Artikel
Sprache:	eng
Schlagworte:	Adult Adult Stem Cells - metabolism Animals Base Sequence Clustered Regularly Interspaced Short Palindromic Repeats - genetics CRISPR-Associated Proteins - metabolism Cystic Fibrosis - metabolism Cystic Fibrosis - pathology Cystic Fibrosis Transmembrane Conductance Regulator - genetics Genetic Therapy Humans Intestines - pathology Mice Molecular Sequence Data Organoids - metabolism RNA Editing - genetics Stem Cells - metabolism
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