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Targeted replacement of full-length CFTR in human airway stem cells by CRISPR-Cas9 for pan-mutation correction in the endogenous locus
Institution:1. Department of Pediatrics, Stanford University, Stanford, CA 94305, USA;2. Department of Internal Medicine, Stanford University, Stanford, CA 94305, USA;3. Department of Otolaryngology–Head and Neck Surgery, Stanford, CA 94305, USA;4. Department of Pathology, Stanford University, Stanford, CA 94305, USA;5. Department of Bioengineering, Rice University, Houston, TX 77030, USA;6. Department of Cell Biology and Physiology, The University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA;7. Department of Psychology, Stanford University, Stanford, CA 94305, USA;8. Department of Biochemistry, Stanford University School of Medicine, Stanford, CA 94305, USA;9. Stanford Cancer Institute, Stanford University School of Medicine, Stanford, CA 94305, USA
Abstract:
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Keywords:cystic fibrosis  airway stem cell therapy  genome editing for CF  CRISPR-Cas9  CFTR correction  CF  universal CFTR correction
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