期刊
JOURNAL OF BIOLOGICAL CHEMISTRY
卷 289, 期 8, 页码 4594-4599出版社
AMER SOC BIOCHEMISTRY MOLECULAR BIOLOGY INC
DOI: 10.1074/jbc.R113.488247
关键词
Embryonic Stem Cell; Gene Therapy; Induced Pluripotent Stem Cell (iPSC); Stem Cells; CRISPR; CAS9; HDAdV; TALEN; Genome Editing; ZFN
资金
- G. Harold and Leila Y. Mathers Charitable Foundation
- Ellison Medical Foundation
- Leona M. and Harry B. Helmsley Charitable Trust
- Glenn Foundation for Medical Research
- California Institute of Regenerative Medicine
- Thousand Young Talents program of China
- National Laboratory of Biomacromolecules
- Strategic Priority Research Program of the Chinese Academy of Sciences
Human pluripotent stem cells (hPSCs) offer unprecedented opportunities to study cellular differentiation and model human diseases. The ability to precisely modify any genomic sequence holds the key to realizing the full potential of hPSCs. Thanks to the rapid development of novel genome editing technologies driven by the enormous interest in the hPSC field, genome editing in hPSCs has evolved from being a daunting task a few years ago to a routine procedure in most laboratories. Here, we provide an overview of the mainstream genome editing tools, including zinc finger nucleases, transcription activator-like effector nucleases, clustered regularly interspaced short palindromic repeat/CAS9 RNA-guided nucleases, and helper-dependent adenoviral vectors. We discuss the features and limitations of these technologies, as well as how these factors influence the utility of these tools in basic research and therapies.
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