Chapter 4 —— 88 —— the LUMC for aiding with the cell sorting procedures and Vincent Mouly (Sorbonne University, Paris, France) for making available the human myoblasts used in this study. Authors in this study are members of the European Reference Network—Neuromuscular Diseases (ERN EURONMD). Author contributions: Z.L. generated and characterized reagents, designed and performed the experiments, examined the datasets and wrote the paper together with M.A.F.V.G.; X.W. performed gene targeting experiments and clonal screens and analyzed the resulting datasets; J.L. generated, characterized and tested reagents; J.M.J. generated, characterized and tested reagents; F.T. setup the DMD gene targeting system; R.H. supervised the research and analyzed the results; M.A.F.V.G. designed and supervised the research, analyzed the data and wrote the paper together with Z.L. Additional Information Data availability: The results supporting this work are available in the article and accompanying supplementary files. The high-throughput deep sequencing library reads are deposited at the NCBI Sequence Read Archive (SRA) database under BioProject ID number: PRJNA1047301. The movies illustrating the viability of human mesenchymal stem cells transduced with second-generation versus high-capacity adenovector particles, the movies illustrating the faster productive transduction kinetics of self-complementary AAV over regular single-stranded AAV vectors, the movies illustrating the impact of CRISPR-induced chromosomal DNA breaks on productive AAV vector transduction, and the movies monitoring the productive AAV transduction kinetics under conditions favoring and not favoring gene knockins are all available through Figshare links indicated in the respective Materials and methods sections. Supplementary data: Supplementary Data are available at doi: 10.1093/nar/gkae1213 Funding: China Scholarship Council - Leiden University Scholarship (to Z.L.
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