The Molecular Biology Society of Japan

[3LBA-124] Targeted Gene Integration in Hematopoietic Stem and Progenitor Cells with Cas9-RNP Method to Universally Correct X-SCID Mutations

〇Suvd Byambaa1, Hideki Uosaki1,2, Hiromasa Hara1,2, Hiroaki Shibata1,2, Tomoyuki Abe1,2, Yasumitsu Nagao3, Osamu Nureki4, Tsukasa Ohmori5, Yutaka Hanazono1,2 (1.Division of Regenerative Medicine, Center for Molecular Medicine, Jichi Medical University, Japan, 2.Center for Development of Advanced Medical Technology, Jichi Medical University, Japan, 3.Center for Experimental Medicine, Jichi Medical University, Japan, 4.Department of Biological Sciences, Graduate School of Science, The University of Tokyo, Japan, 5.Department of Biochemistry, Jichi Medical University, Japan)

Targeted genome-editing therapy, Hematopoietic stem cell, CRISPR/Cas9, Severe combined immunodeficiency, Homology-independent targeted integration

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