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Annu Rev Genomics Hum Genet. 2016 Aug 31;17:131-54. doi: 10.1146/annurev-genom-083115-022258. Epub 2016 May 23.

CRISPR/Cas9 for Human Genome Engineering and Disease Research.

Xiong X1, Chen M2,3,4,5, Lim WA1, Zhao D2, Qi LS2,3,4.

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Department of Cellular and Molecular Pharmacology, University of California, San Francisco, California 94158; email: ,
Department of Bioengineering, Stanford University, Stanford, California 94305; email: ,
Department of Chemical and Systems Biology, Stanford University, Stanford, California 94305.
ChEM-H, Stanford University, Stanford, California 94305.
Gladstone Institute of Cardiovascular Disease, San Francisco, California 94158; email:


The clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated 9 (Cas9) system, a versatile RNA-guided DNA targeting platform, has been revolutionizing our ability to modify, manipulate, and visualize the human genome, which greatly advances both biological research and therapeutics development. Here, we review the current development of CRISPR/Cas9 technologies for gene editing, transcription regulation, genome imaging, and epigenetic modification. We discuss the broad application of this system to the study of functional genomics, especially genome-wide genetic screening, and to therapeutics development, including establishing disease models, correcting defective genetic mutations, and treating diseases.


gene editing; gene regulation; gene therapy; genetic screening; human diseases

[Indexed for MEDLINE]

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