@article{eab09f6a68a445ef94f7394eda473bdd,
title = "Functional repair of CFTR by CRISPR/Cas9 in intestinal stem cell organoids of cystic fibrosis patients",
abstract = "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 response is lost in organoids derived from cystic fibrosis (CF) patients. Here we use the CRISPR/Cas9 genome editing system to correct the CFTR locus by homologous recombination in cultured intestinal stem cells of CF patients. The corrected allele is expressed and fully functional as measured in clonally expanded organoids. This study provides proof of concept for gene correction by homologous recombination in primary adult stem cells derived from patients with a single-gene hereditary defect.",
author = "Gerald Schwank and Koo, {Bon Kyoung} and Valentina Sasselli and Dekkers, {Johanna F.} and Inha Heo and Turan Demircan and Nobuo Sasaki and Sander Boymans and Edwin Cuppen and {Van Der Ent}, {Cornelis K.} and Nieuwenhuis, {Edward E.S.} and Beekman, {Jeffrey M.} and Hans Clevers",
note = "Funding Information: We thank K. Tenbrock (Department of Pediatrics, the RWTH Aachen University, Germany) for providing intestinal rest-material and A. Smith (Wellcome Trust Centre for Stem Cell Research, University of Cambridge) for providing Rosa-CreERT2 mice. This work was funded by grants from the European Research Council (EU/232814-StemCeLLMark), the KNAW/3V-fund, the SNF fellowship for advanced researchers PA00P3 139732 (G.S.), the Human Frontiers in Science Program long-term fellowship LT000422/2012 (G.S.), the Wellcome Trust (097922/C/11/Z) (B.-K.K.), the EU Marie Curie Fellowship EU/330571-FP7-IIF (I.H.), the Astellas Foundation (N.S.), and JSPS (N.S.). ",
year = "2013",
month = dec,
day = "5",
doi = "10.1016/j.stem.2013.11.002",
language = "English",
volume = "13",
pages = "653--658",
journal = "Cell Stem Cell",
issn = "1934-5909",
publisher = "Cell Press",
number = "6",
}