Generation of CF-patient derived iPS cells and efficient footprintless designer nuclease-based gene targeting

*Presenting author The ability to genetically modify human induced pluripotent stem cells (hiPSCs), including the correction of gene defects by means of homologous recombination (HR) is of great interest regarding their potential for ex vivo gene therapy, especially in terms of rare pulmonary diseases like Cystic Fibrosis (CF). Genetic engineering of hiPSCs via customized designer nucleases has been shown to be significantly more efficient than conventional gene targeting, but still typically depends on the introduction of additional genetic selection elements. For the generation of CF patient-specific iPS cells…