A Cut above the Rest: Targeted Genome Editing Technologies in Human Pluripotent Stem Cells*

被引：94

作者：

Li, Mo ^{[1
]}

Suzuki, Keiichiro ^{[1
]}

Kim, Na Young ^{[1
]}

Liu, Guang-Hui ^{[2
]}

Belmonte, Juan Carlos Izpisua ^{[1
]}

机构：

[1] Salk Inst Biol Studies, Gene Express Lab, La Jolla, CA 92037 USA

[2] Chinese Acad Sci, Inst Biophys, Natl Lab Biomacromol, Beijing 100101, Peoples R China

来源：

JOURNAL OF BIOLOGICAL CHEMISTRY | 2014年 / 289卷 / 08期

关键词：

Embryonic Stem Cell; Gene Therapy; Induced Pluripotent Stem Cell (iPSC); Stem Cells; CRISPR; CAS9; HDAdV; TALEN; Genome Editing; ZFN; ZINC-FINGER-NUCLEASES; HOMOLOGOUS RECOMBINATION; GENE CORRECTION; DNA; DISEASE; GENERATION; MUTATIONS; PATIENT; ENDONUCLEASE; SPECIFICITY;

D O I：

10.1074/jbc.R113.488247

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

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.

引用

页码：4594 / 4599

页数：6

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