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Hum Mol Genet. 2018 Aug 1;27(R2):R79-R88. doi: 10.1093/hmg/ddy120.

Development and application of CRISPR/Cas9 technologies in genomic editing.

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Institute of Cell and Development Biology, College of Life Sciences, Zijingang Campus, Zhejiang University, Hangzhou, Zhejiang, P.R. China.
Institute of Orthopedics, Xiaoshan Traditional Chinese Medical Hospital, Hangzhou, Zhejiang, China.


Genomic editing to correct disease-causing mutations is a promising approach for the treatment of human diseases. As a simple and programmable nuclease-based genomic editing tool, the clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) system has substantially improved the ability to make precise changes in the human genome. Rapid development of CRISPR-based technologies in recent years has expanded its application scope and promoted CRISPR-based therapies in preclinical trails. Here, we review the application of the CRISPR system over the last 2 years; including its development and application in base editing, transcription modulation and epigenetic editing, genomic-scale screening, and cell and embryo therapy. Finally, the prospects and challenges related to application of CRISPR/Cas9 technologies are discussed.

[Indexed for MEDLINE]

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