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DNA-PK Inhibition Enhances Gene Editing Efficiency in HSPCs for CRISPR-based Treatment of X-linked Hyper IgM Syndrome

Cole M. Pugliano,Mason Berger,Roslyn M. Ray, Kai Sapkos, Betty Wu, Aidan Laird, Yidian Ye, Daniel Thomson, M. Quinn DeGottardi,Iram F. Khan, Kristina Tatiossian, Brodie A. Miles,Florian Aeschimann, Jerome Pasquier, Mihee M. Kim,David J. Rawlings

MOLECULAR THERAPY METHODS & CLINICAL DEVELOPMENT（2024）

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关键词
CD40L,HPSCs,DNA-PKcs inhibitor,AZD,HDR,CRISPR,AAV6,T cell
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