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Advances in the engineering of the gene editing enzymes and the genomes: Understanding and handling the off-target effects of CRISPR/Cas9

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成果类型:
期刊论文
作者:
Yin, Yufang;Wang, Qian;Xiao, Li*;Wang, Fengjiao;Song, Zhuo;...
通讯作者:
Xiao, Li;Zhang, Jia
作者机构:
[Li, Kai; Liu, Xuan; Yin, Yufang; Zhou, Cuilan; Wang, Qian; Xing, Chungen; Xiao, Li] Soochow Univ, Affiliated Hosp 2, Lab Mol Med, Suzhou 215004, Peoples R China.
[Yin, Yufang] Southern Illinois Univ, Dept Neurosci & Pharmacol, Sch Med, Carbondale, IL 62702 USA.
[Wang, Fengjiao] Soochow Univ, Dept Pharm, Affiliated Childrens Hosp, Suzhou 215123, Peoples R China.
[Song, Zhuo] Genetalks Inc, Changsha 410013, Hunan, Peoples R China.
[He, Nongyue] Southeast Univ, Sch Biol Sci & Med Engn, State Key Lab Bioelect, Nanjing 210096, Jiangsu, Peoples R China.
通讯机构:
[Xiao, Li] S
[Zhang, Jia] U
Soochow Univ, Affiliated Hosp 2, Lab Mol Med, Suzhou 215004, Peoples R China.
Univ South China, Chenzhou Peoples Hosp 1, Translat Med Inst, Natl & Local Joint Engn Lab High Through Mol Diag, Chenzhou 423000, Peoples R China.
语种:
英文
关键词:
Engineering;Gene editing;Gene therapy;Genome
期刊:
JOURNAL OF BIOMEDICAL NANOTECHNOLOGY
ISSN:
1550-7033
年:
2018
卷:
14
期:
3
页码:
456-476
基金类别:
Jiangsu Province's clinical medical science and technology program [BL2013016, QN-868]; Suzhou's Program [SZS201618, SYS201727]; Hunan Province's program [2016JJ2114, 2017SK2172]; National Natural Science Foundation of ChinaNational Natural Science Foundation of China (NSFC) [81301267, 81372825]
机构署名:
本校为通讯机构
院系归属:
医学院
摘要:
In the past decades, significant progresses have been achieved in genetic engineering of nucleases. Among the genetically engineered nucleases, zinc finger nucleases, transcription activator-like (TAL) effector nucleases, and CRIPSPR/Cas9 system form a new field of gene editing. The gene editing efficiency or targeting effect and the off-target effect are the two major determinant factors in evaluating the usefulness of a new enzyme. Engineering strategies in improving these gene editing enzymes, particularly in minimizing their off-target effects, are the focus of this paper. Examples of usin...

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