Zhen Li

Contents Chapter 1 General introduction 9 Chapter 2 “Soft” genome editing using CRISPR nickases as a potential source of safer cell products 15 Chapter 3 AAV-vectored base editor trans-splicing delivers dystrophin repair 31 Chapter 4 Precision genome editing using combinatorial viral vector delivery of CRISPR-Cas9 nucleases and donor DNA constructs 39 Chapter 5 Selector AAV-CRISPR vectors purge off-target chromosomal insertions and promote precise genome editing 97 Chapter 6 Summary and general discussion 151 Chapter 7 Nederlandse Samenvatting Curriculum Vitae List of Publications Acknowledgements 161

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