한빛사 논문
한양대학교, National Institutes of Health
Tae-Hoon Shin1+, Eun Jung Baek1,2+, Marcus A.F. Corat1,3, Shirley Chen1, Jean-Yves Metais1,4, Aisha A. AlJanahi1, Yifan Zhou1, Robert E. Donahue1, Kyung-Rok Yu1,5* , Cynthia E. Dunbar1*
1 Translational Stem Cell Biology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD
2 Department of Laboratory Medicine, College of Medicine, Hanyang University, Seoul, Republic of Korea
3 Multidisciplinary Center for Biological Research, University of Campinas, Campinas, Brazil
4 Department of Bone Marrow Transplantation and Cellular Therapy, St. Jude Children’s Research Hospital, Memphis, TN
5 Department of Medical Life Science, College of Medicine, The Catholic University of Korea, Seoul, Republic of Korea
†These authors contributed equally
*Corresponding authors : Kyung-Rok Yu, Cynthia E. Dunbar
Abstract
Recent advances in gene editing technologies using CRISPR/Cas9 allow precise genome editing at a site of interest and have accelerated human disease modeling and the development of corrective gene therapies for various genetic disorders 1,2. We adapted CRISPR/Cas9 editing of rhesus macaque (RM) hematopoietic stem and progenitor cells HSPCs) to create the first engineered large animal model of a hematologic disease based on close phylogenetic/functional similarity of RM to human HSPCs 3.
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