XB-ART-48565
Trends Biotechnol
2013 Jul 01;317:397-405. doi: 10.1016/j.tibtech.2013.04.004.
Show Gene links
Show Anatomy links
ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering.
Gaj T
,
Gersbach CA
,
Barbas CF
.
???displayArticle.abstract???
Zinc-finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs) comprise a powerful class of tools that are redefining the boundaries of biological research. These chimeric nucleases are composed of programmable, sequence-specific DNA-binding modules linked to a nonspecific DNA cleavage domain. ZFNs and TALENs enable a broad range of genetic modifications by inducing DNA double-strand breaks that stimulate error-prone nonhomologous end joining or homology-directed repair at specific genomic locations. Here, we review achievements made possible by site-specific nuclease technologies and discuss applications of these reagents for genetic analysis and manipulation. In addition, we highlight the therapeutic potential of ZFNs and TALENs and discuss future prospects for the field, including the emergence of clustered regulatory interspaced short palindromic repeat (CRISPR)/Cas-based RNA-guided DNA endonucleases.
???displayArticle.pubmedLink??? 23664777
???displayArticle.pmcLink??? PMC3694601
???displayArticle.link??? Trends Biotechnol
???displayArticle.grants??? [+]
DP1 CA174426 NCI NIH HHS , DP1CA174426 NCI NIH HHS , DP2OD008586 NIH HHS , T32 GM080209 NIGMS NIH HHS , T32GM080209 NIGMS NIH HHS
References [+] :
Bedell,
In vivo genome editing using a high-efficiency TALEN system.
2012, Pubmed
Bedell, In vivo genome editing using a high-efficiency TALEN system. 2012, Pubmed
Beerli, Chemically regulated zinc finger transcription factors. 2000, Pubmed
Beerli, Engineering polydactyl zinc-finger transcription factors. 2002, Pubmed
Beerli, Positive and negative regulation of endogenous genes by designed transcription factors. 2000, Pubmed
Beerli, Toward controlling gene expression at will: specific regulation of the erbB-2/HER-2 promoter by using polydactyl zinc finger proteins constructed from modular building blocks. 1998, Pubmed
Bhakta, Highly active zinc-finger nucleases by extended modular assembly. 2013, Pubmed
Bibikova, Targeted chromosomal cleavage and mutagenesis in Drosophila using zinc-finger nucleases. 2002, Pubmed
Bibikova, Enhancing gene targeting with designed zinc finger nucleases. 2003, Pubmed
Boch, Breaking the code of DNA binding specificity of TAL-type III effectors. 2009, Pubmed
Briggs, Iterative capped assembly: rapid and scalable synthesis of repeat-module DNA such as TAL effectors from individual monomers. 2012, Pubmed
Brunet, Chromosomal translocations induced at specified loci in human stem cells. 2009, Pubmed
Capecchi, Gene targeting in mice: functional analysis of the mammalian genome for the twenty-first century. 2005, Pubmed
Carlson, Efficient TALEN-mediated gene knockout in livestock. 2012, Pubmed
Carroll, Genome engineering with zinc-finger nucleases. 2011, Pubmed
Cermak, Efficient design and assembly of custom TALEN and other TAL effector-based constructs for DNA targeting. 2011, Pubmed
Certo, Coupling endonucleases with DNA end-processing enzymes to drive gene disruption. 2012, Pubmed
Chen, High-frequency genome editing using ssDNA oligonucleotides with zinc-finger nucleases. 2011, Pubmed
Cho, Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease. 2013, Pubmed
Christian, Targeting DNA double-strand breaks with TAL effector nucleases. 2010, Pubmed
Cong, Multiplex genome engineering using CRISPR/Cas systems. 2013, Pubmed
Cristea, In vivo cleavage of transgene donors promotes nuclease-mediated targeted integration. 2013, Pubmed
DeKelver, Functional genomics, proteomics, and regulatory DNA analysis in isogenic settings using zinc finger nuclease-driven transgenesis into a safe harbor locus in the human genome. 2010, Pubmed
Deng, Structural basis for sequence-specific recognition of DNA by TAL effectors. 2012, Pubmed
Ding, A TALEN genome-editing system for generating human stem cell-based disease models. 2013, Pubmed
Doyon, Transient cold shock enhances zinc-finger nuclease-mediated gene disruption. 2010, Pubmed
Doyon, Heritable targeted gene disruption in zebrafish using designed zinc-finger nucleases. 2008, Pubmed
Doyon, Enhancing zinc-finger-nuclease activity with improved obligate heterodimeric architectures. 2011, Pubmed
Ellis, Zinc-finger nuclease-mediated gene correction using single AAV vector transduction and enhancement by Food and Drug Administration-approved drugs. 2013, Pubmed
ENCODE Project Consortium, An integrated encyclopedia of DNA elements in the human genome. 2012, Pubmed
Gabriel, An unbiased genome-wide analysis of zinc-finger nuclease specificity. 2011, Pubmed
Gaj, A comprehensive approach to zinc-finger recombinase customization enables genomic targeting in human cells. 2013, Pubmed
Gaj, Targeted gene knockout by direct delivery of zinc-finger nuclease proteins. 2012, Pubmed
Gaj, Structure-guided reprogramming of serine recombinase DNA sequence specificity. 2011, Pubmed
Gersbach, Targeted plasmid integration into the human genome by an engineered zinc-finger recombinase. 2011, Pubmed
Gersbach, Directed evolution of recombinase specificity by split gene reassembly. 2010, Pubmed
Geurts, Knockout rats via embryo microinjection of zinc-finger nucleases. 2009, Pubmed
Gonzalez, Modular system for the construction of zinc-finger libraries and proteins. 2010, Pubmed
Guo, Directed evolution of an enhanced and highly efficient FokI cleavage domain for zinc finger nucleases. 2010, Pubmed
Gupta, An optimized two-finger archive for ZFN-mediated gene targeting. 2012, Pubmed
Gutschner, Noncoding RNA gene silencing through genomic integration of RNA destabilizing elements using zinc finger nucleases. 2011, Pubmed
Händel, Versatile and efficient genome editing in human cells by combining zinc-finger nucleases with adeno-associated viral vectors. 2012, Pubmed
Hauschild, Efficient generation of a biallelic knockout in pigs using zinc-finger nucleases. 2011, Pubmed
Hockemeyer, Genetic engineering of human pluripotent cells using TALE nucleases. 2011, Pubmed
Hockemeyer, Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases. 2009, Pubmed
Holkers, Differential integrity of TALE nuclease genes following adenoviral and lentiviral vector gene transfer into human cells. 2013, Pubmed
Holt, Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo. 2010, Pubmed
Hwang, Efficient genome editing in zebrafish using a CRISPR-Cas system. 2013, Pubmed
Jiang, RNA-guided editing of bacterial genomes using CRISPR-Cas systems. 2013, Pubmed
Jinek, A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. 2012, Pubmed
Jinek, RNA-programmed genome editing in human cells. 2013, Pubmed
Kim, A library of TAL effector nucleases spanning the human genome. 2013, Pubmed
Kim, Precision genome engineering with programmable DNA-nicking enzymes. 2012, Pubmed
Kim, Getting a handhold on DNA: design of poly-zinc finger proteins with femtomolar dissociation constants. 1998, Pubmed
Kim, Surrogate reporters for enrichment of cells with nuclease-induced mutations. 2011, Pubmed
Kim, Preassembled zinc-finger arrays for rapid construction of ZFNs. 2011, Pubmed
Lee, Targeted chromosomal deletions in human cells using zinc finger nucleases. 2010, Pubmed
Lee, Targeted chromosomal duplications and inversions in the human genome using zinc finger nucleases. 2012, Pubmed
Li, High-efficiency TALEN-based gene editing produces disease-resistant rice. 2012, Pubmed
Li, In vivo genome editing restores haemostasis in a mouse model of haemophilia. 2011, Pubmed
Liu, Design of polydactyl zinc-finger proteins for unique addressing within complex genomes. 1997, Pubmed
Lombardo, Site-specific integration and tailoring of cassette design for sustainable gene transfer. 2011, Pubmed
Lombardo, Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery. 2007, Pubmed
Maeder, Rapid "open-source" engineering of customized zinc-finger nucleases for highly efficient gene modification. 2008, Pubmed
Maeder, Robust, synergistic regulation of human gene expression using TALE activators. 2013, Pubmed
Mak, The crystal structure of TAL effector PthXo1 bound to its DNA target. 2012, Pubmed
Mali, RNA-guided human genome engineering via Cas9. 2013, Pubmed
Maresca, Obligate ligation-gated recombination (ObLiGaRe): custom-designed nuclease-mediated targeted integration through nonhomologous end joining. 2013, Pubmed
McConnell Smith, Generation of a nicking enzyme that stimulates site-specific gene conversion from the I-AniI LAGLIDADG homing endonuclease. 2009, Pubmed
McManus, Gene silencing in mammals by small interfering RNAs. 2002, Pubmed
Meng, Targeted gene inactivation in zebrafish using engineered zinc-finger nucleases. 2008, Pubmed
Mercer, Chimeric TALE recombinases with programmable DNA sequence specificity. 2012, Pubmed
Merlin, Efficient targeted mutagenesis in the monarch butterfly using zinc-finger nucleases. 2013, Pubmed
Miller, A TALE nuclease architecture for efficient genome editing. 2011, Pubmed
Miller, An improved zinc-finger nuclease architecture for highly specific genome editing. 2007, Pubmed
Moehle, Targeted gene addition into a specified location in the human genome using designed zinc finger nucleases. 2007, Pubmed
Morton, Induction and repair of zinc-finger nuclease-targeted double-strand breaks in Caenorhabditis elegans somatic cells. 2006, Pubmed
Moscou, A simple cipher governs DNA recognition by TAL effectors. 2009, Pubmed
Mussolino, A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity. 2011, Pubmed
Neuteboom, Effects of different zinc finger transcription factors on genomic targets. 2006, Pubmed
Orlando, Zinc-finger nuclease-driven targeted integration into mammalian genomes using donors with limited chromosomal homology. 2010, Pubmed
Owens, Chimeric piggyBac transposases for genomic targeting in human cells. 2012, Pubmed
Perez, Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases. 2008, Pubmed
Perez-Pinera, Gene targeting to the ROSA26 locus directed by engineered zinc finger nucleases. 2012, Pubmed
Perez-Pinera, Synergistic and tunable human gene activation by combinations of synthetic transcription factors. 2013, Pubmed
Polstein, Light-inducible spatiotemporal control of gene activation by customizable zinc finger transcription factors. 2012, Pubmed
Provasi, Editing T cell specificity towards leukemia by zinc finger nucleases and lentiviral gene transfer. 2012, Pubmed
Ramirez, Engineered zinc finger nickases induce homology-directed repair with reduced mutagenic effects. 2012, Pubmed
Reyon, FLASH assembly of TALENs for high-throughput genome editing. 2012, Pubmed
Sander, Selection-free zinc-finger-nuclease engineering by context-dependent assembly (CoDA). 2011, Pubmed
Sander, Targeted gene disruption in somatic zebrafish cells using engineered TALENs. 2011, Pubmed
Santiago, Targeted gene knockout in mammalian cells by using engineered zinc-finger nucleases. 2008, Pubmed
Sanyal, The long-range interaction landscape of gene promoters. 2012, Pubmed
Schmid-Burgk, A ligation-independent cloning technique for high-throughput assembly of transcription activator–like effector genes. 2013, Pubmed
Sebastiano, In situ genetic correction of the sickle cell anemia mutation in human induced pluripotent stem cells using engineered zinc finger nucleases. 2011, Pubmed
Segal, Toward controlling gene expression at will: selection and design of zinc finger domains recognizing each of the 5'-GNN-3' DNA target sequences. 1999, Pubmed
Segal, Structure of Aart, a designed six-finger zinc finger peptide, bound to DNA. 2006, Pubmed
Shukla, Precise genome modification in the crop species Zea mays using zinc-finger nucleases. 2009, Pubmed
Soldner, Generation of isogenic pluripotent stem cells differing exclusively at two early onset Parkinson point mutations. 2011, Pubmed
Söllü, Autonomous zinc-finger nuclease pairs for targeted chromosomal deletion. 2010, Pubmed
Sood, Efficient methods for targeted mutagenesis in zebrafish using zinc-finger nucleases: data from targeting of nine genes using CompoZr or CoDA ZFNs. 2013, Pubmed
Szczepek, Structure-based redesign of the dimerization interface reduces the toxicity of zinc-finger nucleases. 2007, Pubmed
Tesson, Knockout rats generated by embryo microinjection of TALENs. 2011, Pubmed
Torikai, A foundation for universal T-cell based immunotherapy: T cells engineered to express a CD19-specific chimeric-antigen-receptor and eliminate expression of endogenous TCR. 2012, Pubmed
Townsend, High-frequency modification of plant genes using engineered zinc-finger nucleases. 2009, Pubmed
Urnov, Genome editing with engineered zinc finger nucleases. 2010, Pubmed
Urnov, Highly efficient endogenous human gene correction using designed zinc-finger nucleases. 2005, Pubmed
Voit, Generation of an HIV resistant T-cell line by targeted "stacking" of restriction factors. 2013, Pubmed
Wang, Targeted gene addition to a predetermined site in the human genome using a ZFN-based nicking enzyme. 2012, Pubmed
Wiedenheft, RNA-guided genetic silencing systems in bacteria and archaea. 2012, Pubmed
Wood, Targeted genome editing across species using ZFNs and TALENs. 2011, Pubmed
Wyman, DNA double-strand break repair: all's well that ends well. 2006, Pubmed
Yant, Site-directed transposon integration in human cells. 2007, Pubmed
Young, Efficient targeted gene disruption in the soma and germ line of the frog Xenopus tropicalis using engineered zinc-finger nucleases. 2011, Pubmed , Xenbase
Yusa, Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells. 2011, Pubmed
Zhang, Efficient construction of sequence-specific TAL effectors for modulating mammalian transcription. 2011, Pubmed
Zhang, High frequency targeted mutagenesis in Arabidopsis thaliana using zinc finger nucleases. 2010, Pubmed
Zou, Site-specific gene correction of a point mutation in human iPS cells derived from an adult patient with sickle cell disease. 2011, Pubmed
Zou, Gene targeting of a disease-related gene in human induced pluripotent stem and embryonic stem cells. 2009, Pubmed
Zu, TALEN-mediated precise genome modification by homologous recombination in zebrafish. 2013, Pubmed