Isolation of CF cell lines corrected at ΔF508-CFTR locus by SFHR-mediated targeting

  title={Isolation of CF cell lines corrected at ΔF508-CFTR locus by SFHR-mediated targeting},
  author={Emanuela Bruscia and Federica Carla Sangiuolo and Paola Sinibaldi and Kaarin K. Goncz and Giuseppe Novelli and Dieter C. Gruenert},
  journal={Gene Therapy},
Cystic fibrosis is the most common inherited disease in the Caucasian population. About 70% of all CF chromosomes carry the ΔF508 mutation, a 3-bp deletion that results in the loss of a phenylalanine at amino acid 508 in the CF transmembrane conductance regulator (CFTR) protein. Direct modification of the ΔF508 locus of endogenous CFTR was achieved by small fragment homologous replacement (SFHR). Transformed human airway epithelial cells (CFBE41o−), homozygous for ΔF508 mutation, were… CONTINUE READING
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