Targeted genome editing using engineered nucleases has rapidly gone from being a niche technology to a mainstream method used by many biological researchers. This widespread adoption has been largely fueled by the emergence of the clustered, regularly interspaced, short palindromic repeat (CRISPR) technology, an important new approach for generating RNA-guided nucleases, such as Cas9, with customizable specificities. Genome editing mediated by these nucleases has been used to rapidly, easily and efficiently modify endogenous genes in a wide variety of biomedically important cell types and in organisms that have traditionally been challenging to manipulate genetically. Furthermore, a modified version of the CRISPR-Cas9 system has been developed to recruit heterologous domains that can regulate endogenous gene expression or label specific genomic loci in living cells. Although the genome-wide specificities of CRISPR-Cas9 systems remain to be fully defined, the power of these systems to perform targeted, highly efficient alterations of genome sequence and gene expression will undoubtedly transform biological research and spur the development of novel molecular therapeutics for human disease.
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J.K.J. is grateful for support from the US National Institutes of Health (NIH) (grants DP1 GM105378 and R01 GM088040), the Defense Advanced Research Projects Agency (grant W911NF-11-2-0056) and The Jim and Ann Orr Massachusetts General Hospital Research Scholar Award. This material is based upon work supported fully or in part by the US Army Research Laboratory and the US Army Research Office under grant number W911NF-11-2-0056. The authors apologize to colleagues whose studies were not cited due to length and reference constraints.
J.K.J. has financial interests in Editas Medicine and Transposagen Biopharmaceuticals. J.K.J.'s interests were reviewed and are managed by Massachusetts General Hospital and Partners HealthCare in accordance with their conflict of interest policies. J.K.J. and J.D.S. are consultants for Editas Medicine.
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Sander, J., Joung, J. CRISPR-Cas systems for editing, regulating and targeting genomes. Nat Biotechnol 32, 347–355 (2014). https://doi.org/10.1038/nbt.2842
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