Development and potential applications of CRISPR-Cas9 genome editing technology in sarcoma

被引：32
作者：
Liu, Tang [1 ,2 ,3 ]
Shen, Jacson K. [1 ,2 ]
Li, Zhihong [3 ]
Choy, Edwin [1 ,2 ]
Hornicek, Francis J. [1 ,2 ]
Duan, Zhenfeng [1 ,2 ]
机构：
[1] Massachusetts Gen Hosp, Dept Orthopaed Surg, Sarcoma Biol Lab, 55 Fruit St,Jackson 1115, Boston, MA 02114 USA
[2] Harvard Univ, Sch Med, 55 Fruit St,Jackson 1115, Boston, MA 02114 USA
[3] Cent South Univ, Xiangya Hosp 2, Dept Orthopaed, 139 Renmin Rd, Changsha 410011, Hunan, Peoples R China
来源：
CANCER LETTERS | 2016年 / 373卷 / 01期
基金：
美国国家卫生研究院;
关键词：
CRISPR-Cas9; Sarcoma; Cancer modeling; Gene therapy; MOUSE MODEL; CHROMOSOMAL TRANSLOCATIONS; ANTITUMOR-ACTIVITY; CELLULAR-ORIGINS; NUCLEAR EXPORT; CANCER; P53; CRISPR/CAS9; TUMOR; EXPRESSION;
D O I：
10.1016/j.canlet.2016.01.030
中图分类号：
R73 [肿瘤学];
学科分类号：
100214 ;
摘要：
Sarcomas include some of the most aggressive tumors and typically respond poorly to chemotherapy. In recent years, specific gene fusion/mutations and gene over-expression/activation have been shown to drive sarcoma pathogenesis and development. These emerging genomic alterations may provide targets for novel therapeutic strategies and have the potential to transform sarcoma patient care. The RNA guided nuclease CRISPR-Cas9 (Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-associated protein-9 nuclease) is a convenient and versatile platform for site-specific genome editing and epigenome targeted modulation. Given that sarcoma is believed to develop as a result of genetic alterations in mesenchymal progenitor/stem cells, CRISPR-Cas9 genome editing technologies hold extensive application potentials in sarcoma models and therapies. We review the development and mechanisms of the CRISPR-Cas9 system in genome editing and introduce its application in sarcoma research and potential therapy in clinic. Additionally, we propose future directions and discuss the challenges faced with these applications, providing concise and enlightening information for readers interested in this area. (C) 2016 Elsevier Ireland Ltd. All rights reserved.
引用
收藏
页码：109 / 118
页数：10
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