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Potential pitfalls of CRISPR/Cas9-mediated genome editing.

Content Provider	World Health Organization (WHO)-Global Index Medicus
Author	Peng, Rongxue Lin, Guigao Li, Jinming
Description	Country affiliation: China Author Affiliation: Peng R ( National Center for Clinical Laboratories, Beijing Hospital, Beijing, China.); Lin G ( Graduate School, Peking Union Medical College, Chinese Academy of Medical Sciences, Beijing, China.); Li J ( National Center for Clinical Laboratories, Beijing Hospital, Beijing, China.)
Abstract	Recently, a novel technique named the clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated protein (Cas)9 system has been rapidly developed. This genome editing tool has improved our ability tremendously with respect to exploring the pathogenesis of diseases and correcting disease mutations, as well as phenotypes. With a short guide RNA, Cas9 can be precisely directed to target sites, and functions as an endonuclease to efficiently produce breaks in DNA double strands. Over the past 30 years, CRISPR has evolved from the 'curious sequences of unknown biological function' into a promising genome editing tool. As a result of the incessant development in the CRISPR/Cas9 system, Cas9 co-expressed with custom guide RNAs has been successfully used in a variety of cells and organisms. This genome editing technology can also be applied to synthetic biology, functional genomic screening, transcriptional modulation and gene therapy. However, although CRISPR/Cas9 has a broad range of action in science, there are several aspects that affect its efficiency and specificity, including Cas9 activity, target site selection and short guide RNA design, delivery methods, off-target effects and the incidence of homology-directed repair. In the present review, we highlight the factors that affect the utilization of CRISPR/Cas9, as well as possible strategies for handling any problems. Addressing these issues will allow us to take better advantage of this technique. In addition, we also review the history and rapid development of the CRISPR/Cas system from the time of its initial discovery in 2012.
File Format	HTM / HTML
ISSN	1742464X
Issue Number	7
Volume Number	283
e-ISSN	17424658
Journal	FEBS Journal
Language	English
Publisher	Wiley (on behalf of the Federation of European Biochemical Societies)
Publisher Date	2016-04-01
Publisher Place	Great Britain (UK)
Access Restriction	One Nation One Subscription (ONOS)
Subject Keyword	Discipline Biochemistry Crispr-cas Systems Gene Targeting Methods Genetic Engineering Genetic Therapy Genome, Human Genetics Binding Sites Humans Models, Genetic Rna, Guide Reproducibility Of Results Journal Article Research Support, Non-u.s. Gov't Review
Content Type	Text
Resource Type	Article
Subject	Cell Biology Biochemistry Molecular Biology

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