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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.

Author information

1
Institute of Cell and Development Biology, College of Life Sciences, Zijingang Campus, Zhejiang University, Hangzhou, Zhejiang, P.R. China.
2
Institute of Orthopedics, Xiaoshan Traditional Chinese Medical Hospital, Hangzhou, Zhejiang, China.

Abstract

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.

PMID:
29659822
DOI:
10.1093/hmg/ddy120
[Indexed for MEDLINE]

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