Advances and Obstacles in Homology-Mediated Gene Editing of Hematopoietic Stem Cells.
Advances and Obstacles in Homology-Mediated Gene Editing of Hematopoietic Stem Cells.
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DOI：
10.3390/jcm10030513
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发表时间：
2021-02-01
影响因子：
3.9
通讯作者：
Larochelle A
中科院分区：
文献类型：
--
作者：
Salisbury-Ruf CT;Larochelle A
Homology-directed gene editing of hematopoietic stem and progenitor cells (HSPCs) is a promising strategy for the treatment of inherited blood disorders, obviating many of the limitations associated with viral vector-mediated gene therapies. The use of CRISPR/Cas9 or other programmable nucleases and improved methods of homology template delivery have enabled precise ex vivo gene editing. These transformative advances have also highlighted technical challenges to achieve high-efficiency gene editing in HSPCs for therapeutic applications. In this review, we discuss recent pre-clinical investigations utilizing homology-mediated gene editing in HSPCs and highlight various strategies to improve editing efficiency in these cells.
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影响因子：
5.9
作者：
Brown A;Pospiech J;Eiwen K;Baker DJ;Moehrle B;Sakk V;Nattamai K;Vogel M;Grigoryan A;Geiger H
通讯作者：
Geiger H
影响因子：
16.6
作者：
Carlson-Stevermer J;Abdeen AA;Kohlenberg L;Goedland M;Molugu K;Lou M;Saha K
通讯作者：
Saha K
影响因子：
14.8
作者：
Bak RO;Dever DP;Porteus MH
通讯作者：
Porteus MH
影响因子：
15.1
作者：
Chou, Shih-Jie;Yang, Peng;Chiou, Shih-Hwa
通讯作者：
Chiou, Shih-Hwa
DOI：
10.1073/pnas.1818461116
发表时间：
2019-10-15
影响因子：
11.1
作者：
Acharya, Sundaram;Mishra, Arpit;Chakraborty, Debojyoti
通讯作者：
Chakraborty, Debojyoti