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      CRISPR RNA-guided activation of endogenous human genes

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          Abstract

          Catalytically inactive CRISPR-associated 9 nuclease (dCas9) can be directed by short guide RNAs (gRNAs) to repress endogenous genes in bacteria and human cells. Here we show that a dCas9-VP64 transcriptional activation domain fusion protein can be directed by single or multiple gRNAs to increase expression of specific endogenous human genes. These results provide an important proof-of-principle that CRISPR-Cas systems can be used to target heterologous effector domains in human cells.

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          Most cited references 7

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          Efficient In Vivo Genome Editing Using RNA-Guided Nucleases

          Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas) systems have evolved in bacteria and archaea as a defense mechanism to silence foreign nucleic acids of viruses and plasmids. Recent work has shown that bacterial type II CRISPR systems can be adapted to create guide RNAs (gRNAs) capable of directing site-specific DNA cleavage by the Cas9 nuclease in vitro. Here we show that this system can function in vivo to induce targeted genetic modifications in zebrafish embryos with efficiencies comparable to those obtained using ZFNs and TALENs for the same genes. RNA-guided nucleases robustly enabled genome editing at 9 of 11 different sites tested, including two for which TALENs previously failed to induce alterations. These results demonstrate that programmable CRISPR/Cas systems provide a simple, rapid, and highly scalable method for altering genes in vivo, opening the door to using RNA-guided nucleases for genome editing in a wide range of organisms.
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            Generation of gene-modified mice via Cas9/RNA-mediated gene targeting.

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              Enhanced efficiency of human pluripotent stem cell genome editing through replacing TALENs with CRISPRs.

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                Author and article information

                Journal
                101215604
                32338
                Nat Methods
                Nat. Methods
                Nature methods
                1548-7091
                1548-7105
                16 September 2013
                25 July 2013
                October 2013
                01 April 2014
                : 10
                : 10
                : 977-979
                Affiliations
                [1 ]Molecular Pathology Unit, Massachusetts General Hospital, Charlestown, MA 02129 USA
                [2 ]Center for Computational and Integrative Biology, Massachusetts General Hospital, Charlestown, MA 02129 USA
                [3 ]Center for Cancer Research, Massachusetts General Hospital, Charlestown, MA 02129 USA
                [4 ]Program in Biological and Biomedical Sciences, Harvard Medical School, Boston, MA 02115 USA
                [5 ]Department of Pathology, Harvard Medical School, Boston, MA 02115 USA
                Author notes
                Correspondence to: JJOUNG@ 123456PARTNERS.ORG
                Article
                NIHMS508103
                10.1038/nmeth.2598
                3794058
                23892898

                Users may view, print, copy, download and text and data- mine the content in such documents, for the purposes of academic research, subject always to the full Conditions of use: http://www.nature.com/authors/editorial_policies/license.html#terms

                Funding
                Funded by: National Institute of Neurological Disorders and Stroke : NINDS
                Award ID: R01 NS073124 || NS
                Funded by: National Human Genome Research Institute : NHGRI
                Award ID: P50 HG005550 || HG
                Funded by: National Institute of General Medical Sciences : NIGMS
                Award ID: DP1 GM105378 || GM
                Categories
                Article

                Life sciences

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