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

Cell stem cell · 2013 · Vol. 13(6) · pp. 653–658

Gerald Schwank(University Medical Center Utrecht)Bon‐Kyoung Koo(University Medical Center Utrecht)Valentina Sasselli(University Medical Center Utrecht)Johanna F. Dekkers(Wilhelmina Children's Hospital)Inha Heo(University Medical Center Utrecht)Turan Demircan(Hubrecht Institute for Developmental Biology and Stem Cell Research)Nobuo Sasaki(University Medical Center Utrecht)Sander Boymans(Hubrecht Institute for Developmental Biology and Stem Cell Research)Edwin Cuppen(University Medical Center Utrecht)Cornelis K. van der Ent(Wilhelmina Children's Hospital)Edward E. S. Nieuwenhuis(Wilhelmina Children's Hospital)Jeffrey M. Beekman(University Medical Center Utrecht)Hans Clevers✉(University Medical Center Utrecht)

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Pluripotent Stem Cells Research CRISPR and Genetic Engineering Cystic Fibrosis Research AdvancesOrganoidBiologyCRISPRCystic fibrosisStem cellGenome editingHomologous recombinationCas9Cell biologyCystic fibrosis transmembrane conductance regulator

MeSH terms

AdultAnimalsBase SequenceCystic FibrosisHumansIntestinesMolecular Sequence DataOrganoidsStem CellsGenetic TherapyRNA EditingCystic Fibrosis Transmembrane Conductance RegulatorMiceAdult Stem CellsClustered Regularly Interspaced Short Palindromic Repeats

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