[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 Uosaki^1,2, Hiromasa Hara^1,2, Hiroaki Shibata^1,2, Tomoyuki Abe^1,2, Yasumitsu Nagao³, Osamu Nureki⁴, Tsukasa Ohmori⁵, Yutaka Hanazono^1,2 (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