Delivery technologies for genome editing

被引：410
|
作者：
Yin, Hao [1 ]
Kauffman, Kevin J. [1 ,2 ]
Anderson, Daniel G. [1 ,2 ,3 ,4 ]
机构：
[1] MIT, David H Koch Inst Integrat Canc Res, Cambridge, MA 02139 USA
[2] MIT, Dept Chem Engn, Cambridge, MA 02139 USA
[3] Harvard Massachusetts Inst Technol, Div Hlth Sci & Technol, Cambridge, MA 02139 USA
[4] MIT, Inst Med Engn & Sci, Cambridge, MA 02139 USA
来源：
NATURE REVIEWS DRUG DISCOVERY | 2017年 / 16卷 / 06期
关键词：
ZINC-FINGER NUCLEASES; HEMATOPOIETIC STEM-CELLS; HEMOPHILIA GENE-THERAPY; DOUBLE-STRAND BREAKS; OFF-TARGET CLEAVAGE; HUMAN T-CELLS; IN-VIVO; MESSENGER-RNA; REPLACEMENT THERAPY; MUSCULAR-DYSTROPHY;
D O I：
10.1038/nrd.2016.280
中图分类号：
Q81 [生物工程学（生物技术）]; Q93 [微生物学];
学科分类号：
071005 ; 0836 ; 090102 ; 100705 ;
摘要：
With the recent development of CRISPR technology, it is becoming increasingly easy to engineer the genome. Genome-editing systems based on CRISPR, as well as transcription activator-like effector nucleases (TALENs) and zinc-finger nucleases (ZFNs), are becoming valuable tools for biomedical research, drug discovery and development, and even gene therapy. However, for each of these systems to effectively enter cells of interest and perform their function, efficient and safe delivery technologies are needed. This Review discusses the principles of biomacromolecule delivery and gene editing, examines recent advances and challenges in non-viral and viral delivery methods, and highlights the status of related clinical trials.
引用
收藏
页码：387 / 399
页数：13
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