期刊
HUMAN MOLECULAR GENETICS
卷 27, 期 R2, 页码 R79-R88出版社
OXFORD UNIV PRESS
DOI: 10.1093/hmg/ddy120
关键词
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资金
- Chinese National Nature Science Foundation [81570932, U1738102]
- National Basic Research Program of China [2014CB541705]
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.
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