Highly Efficient Genome Editing of Murine and Human Hematopoietic Progenitor Cells by CRISPR/Cas9
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- 作者：Michael C. Gundry1 ; 2 ; 5 ; 10 ; Lorenzo Brunetti2 ; 5 ; 7 ; 10 ; Angelique Lin1 ; 3 ; 10 ; Allison E. Mayle1 ; 2 ; 5 ; Ayumi Kitano1 ; Dimitrios Wagner5 ; 8 ; 9 ; Joanne I. Hsu2 ; 4 ; 5 ; Kevin A. Hoegenauer1 ; Cliona M. Rooney5 ; 6 ; 8 ; Margaret A. Goodell1 ; 2 ; 5 ; 6 ; 8 ; goodell@bcm.edu" class="auth_mail" title="E-mail the corresponding author ; Daisuke Nakada1 ; 2 ; 5 ; 11 ; nakada@bcm.edu" class="auth_mail" title="E-mail the corresponding author
- 关键词：HSC ; hematopoietic stem cells ; progenitor ; human CD34 ; genome editing ; CRISPR/Cas9 ; sgRNA ; homology-directed repair ; gene therapy ; transplantation
- 刊名：Cell Reports
- 出版年：2016
- 出版时间：25 October 2016
- 年：2016
- 卷：17
- 期：5
- 页码：1453-1461
- 全文大小：2425 K
文摘
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Gene knockout of primary murine HSPCs with efficiencies routinely higher than 60%
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Gene knockout of primary human HSPCs and T cells from 75% to 90%
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CRISPR/Cas9 homology-directed repair in >20% of primary human HSPCs