Published in Nat Biotechnol on August 07, 2011
High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells. Nat Biotechnol (2013) 18.01
Development and applications of CRISPR-Cas9 for genome engineering. Cell (2014) 13.50
FLASH assembly of TALENs for high-throughput genome editing. Nat Biotechnol (2012) 12.97
ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering. Trends Biotechnol (2013) 11.58
CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering. Nat Biotechnol (2013) 11.35
Analysis of off-target effects of CRISPR/Cas-derived RNA-guided endonucleases and nickases. Genome Res (2013) 6.19
GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases. Nat Biotechnol (2014) 5.69
CRISPR/Cas9 systems targeting β-globin and CCR5 genes have substantial off-target activity. Nucleic Acids Res (2013) 4.11
Highly efficient generation of heritable zebrafish gene mutations using homo- and heterodimeric TALENs. Nucleic Acids Res (2012) 3.41
Highly efficient RNA-guided genome editing in human cells via delivery of purified Cas9 ribonucleoproteins. Genome Res (2014) 3.31
Precision genome engineering with programmable DNA-nicking enzymes. Genome Res (2012) 3.05
Genome-wide detection of DNA double-stranded breaks induced by engineered nucleases. Nat Biotechnol (2014) 2.99
A library of TAL effector nucleases spanning the human genome. Nat Biotechnol (2013) 2.87
Targeted chromosomal duplications and inversions in the human genome using zinc finger nucleases. Genome Res (2011) 2.85
Targeted genome editing in human repopulating haematopoietic stem cells. Nature (2014) 2.49
Therapeutic genome editing: prospects and challenges. Nat Med (2015) 2.42
RNA-directed gene editing specifically eradicates latent and prevents new HIV-1 infection. Proc Natl Acad Sci U S A (2014) 2.32
Engineered zinc finger nickases induce homology-directed repair with reduced mutagenic effects. Nucleic Acids Res (2012) 2.26
Targeted gene correction minimally impacts whole-genome mutational load in human-disease-specific induced pluripotent stem cell clones. Cell Stem Cell (2014) 2.24
Obligate ligation-gated recombination (ObLiGaRe): custom-designed nuclease-mediated targeted integration through nonhomologous end joining. Genome Res (2012) 2.12
Targeted gene knockout by direct delivery of zinc-finger nuclease proteins. Nat Methods (2012) 2.07
Unbiased detection of off-target cleavage by CRISPR-Cas9 and TALENs using integrase-defective lentiviral vectors. Nat Biotechnol (2015) 2.03
Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells. Blood (2015) 2.00
Advances in targeted genome editing. Curr Opin Chem Biol (2012) 1.97
Gene therapy returns to centre stage. Nature (2015) 1.95
Dissecting neural function using targeted genome engineering technologies. ACS Chem Neurosci (2012) 1.89
TALEN-based gene correction for epidermolysis bullosa. Mol Ther (2013) 1.84
RNA guides genome engineering. Nat Biotechnol (2013) 1.84
Recombinant probes for visualizing endogenous synaptic proteins in living neurons. Neuron (2013) 1.80
Reducing ligation bias of small RNAs in libraries for next generation sequencing. Silence (2012) 1.68
Broad specificity profiling of TALENs results in engineered nucleases with improved DNA-cleavage specificity. Nat Methods (2014) 1.66
Quantifying genome-editing outcomes at endogenous loci with SMRT sequencing. Cell Rep (2014) 1.50
Genomic editing of the HIV-1 coreceptor CCR5 in adult hematopoietic stem and progenitor cells using zinc finger nucleases. Mol Ther (2013) 1.49
A cut above the rest: targeted genome editing technologies in human pluripotent stem cells. J Biol Chem (2013) 1.47
Efficient correction of hemoglobinopathy-causing mutations by homologous recombination in integration-free patient iPSCs. Cell Res (2011) 1.47
In silico abstraction of zinc finger nuclease cleavage profiles reveals an expanded landscape of off-target sites. Nucleic Acids Res (2013) 1.46
An online bioinformatics tool predicts zinc finger and TALE nuclease off-target cleavage. Nucleic Acids Res (2013) 1.45
Genome-editing Technologies for Gene and Cell Therapy. Mol Ther (2016) 1.41
Making designer mutants in model organisms. Development (2014) 1.38
Genome editing of human pluripotent stem cells to generate human cellular disease models. Dis Model Mech (2013) 1.37
Precision editing of large animal genomes. Adv Genet (2012) 1.34
Retroviral integrations in gene therapy trials. Mol Ther (2012) 1.34
Hematopoietic-stem-cell-based gene therapy for HIV disease. Cell Stem Cell (2012) 1.33
megaTALs: a rare-cleaving nuclease architecture for therapeutic genome engineering. Nucleic Acids Res (2013) 1.30
Target specificity of the CRISPR-Cas9 system. Quant Biol (2014) 1.23
Off-target Effects in CRISPR/Cas9-mediated Genome Engineering. Mol Ther Nucleic Acids (2015) 1.23
Type II restriction endonucleases--a historical perspective and more. Nucleic Acids Res (2014) 1.22
Bioinformatic clonality analysis of next-generation sequencing-derived viral vector integration sites. Hum Gene Ther Methods (2012) 1.21
Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors. Nat Biotechnol (2015) 1.17
TALENs facilitate targeted genome editing in human cells with high specificity and low cytotoxicity. Nucleic Acids Res (2014) 1.16
Zinc-finger nuclease-mediated correction of α-thalassemia in iPS cells. Blood (2012) 1.15
Quantifying on- and off-target genome editing. Trends Biotechnol (2015) 1.15
Efficient clinical scale gene modification via zinc finger nuclease-targeted disruption of the HIV co-receptor CCR5. Hum Gene Ther (2013) 1.14
A TALE of two nucleases: gene targeting for the masses? Zebrafish (2011) 1.14
Expanding the genetic editing tool kit: ZFNs, TALENs, and CRISPR-Cas9. J Clin Invest (2014) 1.13
Targeting DNA With Fingers and TALENs. Mol Ther Nucleic Acids (2012) 1.12
TALEN-mediated single-base-pair editing identification of an intergenic mutation upstream of BUB1B as causative of PCS (MVA) syndrome. Proc Natl Acad Sci U S A (2013) 1.11
Robust ZFN-mediated genome editing in adult hemophilic mice. Blood (2013) 1.11
Hematopoietic stem cell engineering at a crossroads. Blood (2011) 1.10
Gene therapy on the move. EMBO Mol Med (2013) 1.10
Correction of dystrophin expression in cells from Duchenne muscular dystrophy patients through genomic excision of exon 51 by zinc finger nucleases. Mol Ther (2014) 1.09
Chimeric piggyBac transposases for genomic targeting in human cells. Nucleic Acids Res (2012) 1.09
Highly significant antiviral activity of HIV-1 LTR-specific tre-recombinase in humanized mice. PLoS Pathog (2013) 1.08
Zinc-finger nucleases: how to play two good hands. Nat Methods (2011) 1.08
Newer gene editing technologies toward HIV gene therapy. Viruses (2013) 1.07
Induced Pluripotent Stem Cells Meet Genome Editing. Cell Stem Cell (2016) 1.06
Compact designer TALENs for efficient genome engineering. Nat Commun (2013) 1.06
GT-Scan: identifying unique genomic targets. Bioinformatics (2014) 1.05
Evaluation of TCR Gene Editing Achieved by TALENs, CRISPR/Cas9, and megaTAL Nucleases. Mol Ther (2015) 1.04
Versatile and efficient genome editing in human cells by combining zinc-finger nucleases with adeno-associated viral vectors. Hum Gene Ther (2011) 1.04
AAV-mediated delivery of zinc finger nucleases targeting hepatitis B virus inhibits active replication. PLoS One (2014) 1.04
Defining and improving the genome-wide specificities of CRISPR-Cas9 nucleases. Nat Rev Genet (2016) 1.03
Targeted gene addition in human CD34(+) hematopoietic cells for correction of X-linked chronic granulomatous disease. Nat Biotechnol (2016) 1.03
Chromatin regulation at the frontier of synthetic biology. Nat Rev Genet (2015) 1.01
Transcription activator like effector (TALE)-directed piggyBac transposition in human cells. Nucleic Acids Res (2013) 1.01
High-efficiency genome editing via 2A-coupled co-expression of fluorescent proteins and zinc finger nucleases or CRISPR/Cas9 nickase pairs. Nucleic Acids Res (2014) 0.98
Seamless genome editing in human pluripotent stem cells using custom endonuclease-based gene targeting and the piggyBac transposon. Nat Protoc (2013) 0.97
Measuring and Reducing Off-Target Activities of Programmable Nucleases Including CRISPR-Cas9. Mol Cells (2015) 0.97
Creating and evaluating accurate CRISPR-Cas9 scalpels for genomic surgery. Nat Methods (2015) 0.96
CCR5 Gene Editing of Resting CD4(+) T Cells by Transient ZFN Expression From HIV Envelope Pseudotyped Nonintegrating Lentivirus Confers HIV-1 Resistance in Humanized Mice. Mol Ther Nucleic Acids (2014) 0.96
Determining the specificities of TALENs, Cas9, and other genome-editing enzymes. Methods Enzymol (2014) 0.93
Find and replace: editing human genome in pluripotent stem cells. Protein Cell (2011) 0.93
Generation and genetic engineering of human induced pluripotent stem cells using designed zinc finger nucleases. Stem Cells Dev (2012) 0.93
A novel zinc-finger nuclease platform with a sequence-specific cleavage module. Nucleic Acids Res (2011) 0.92
Expanding the scope of site-specific recombinases for genetic and metabolic engineering. Biotechnol Bioeng (2013) 0.92
A systematic survey of the Cys2His2 zinc finger DNA-binding landscape. Nucleic Acids Res (2015) 0.92
How specific is CRISPR/Cas9 really? Curr Opin Chem Biol (2015) 0.91
Targeted gene therapies: tools, applications, optimization. Crit Rev Biochem Mol Biol (2012) 0.91
mRNA transfection of a novel TAL effector nuclease (TALEN) facilitates efficient knockout of HIV co-receptor CCR5. Nucleic Acids Res (2015) 0.91
Directed evolution of a recombinase that excises the provirus of most HIV-1 primary isolates with high specificity. Nat Biotechnol (2016) 0.90
EENdb: a database and knowledge base of ZFNs and TALENs for endonuclease engineering. Nucleic Acids Res (2012) 0.90
Enabling functional genomics with genome engineering. Genome Res (2015) 0.90
Assessing the risks of genotoxicity in the therapeutic development of induced pluripotent stem cells. Mol Ther (2012) 0.90
Progress and prospects in stem cell therapy. Acta Pharmacol Sin (2013) 0.90
Transcriptional repression of Gata3 is essential for early B cell commitment. Immunity (2013) 0.89
Genome editing and the next generation of antiviral therapy. Hum Genet (2016) 0.88
Precise gene modification mediated by TALEN and single-stranded oligodeoxynucleotides in human cells. PLoS One (2014) 0.88
Creating highly specific nucleases by fusion of active restriction endonucleases and catalytically inactive homing endonucleases. Nucleic Acids Res (2011) 0.87
Fanconi anemia gene editing by the CRISPR/Cas9 system. Hum Gene Ther (2015) 0.87
Gene- and protein-delivered zinc finger-staphylococcal nuclease hybrid for inhibition of DNA replication of human papillomavirus. PLoS One (2013) 0.87
Basic local alignment search tool. J Mol Biol (1990) 659.07
BLAT--the BLAST-like alignment tool. Genome Res (2002) 126.78
In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science (1996) 28.95
Highly efficient endogenous human gene correction using designed zinc-finger nucleases. Nature (2005) 19.15
Genome editing with engineered zinc finger nucleases. Nat Rev Genet (2010) 18.88
An improved zinc-finger nuclease architecture for highly specific genome editing. Nat Biotechnol (2007) 17.80
Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases. Nat Biotechnol (2008) 14.75
HIV-1 integration in the human genome favors active genes and local hotspots. Cell (2002) 12.60
Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases. Nat Biotechnol (2009) 12.40
Knockout rats via embryo microinjection of zinc-finger nucleases. Science (2009) 12.33
Hybrid restriction enzymes: zinc finger fusions to Fok I cleavage domain. Proc Natl Acad Sci U S A (1996) 12.25
Rapid "open-source" engineering of customized zinc-finger nucleases for highly efficient gene modification. Mol Cell (2008) 11.24
Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery. Nat Biotechnol (2007) 9.23
Targeted chromosomal cleavage and mutagenesis in Drosophila using zinc-finger nucleases. Genetics (2002) 9.09
Distinct factors control histone variant H3.3 localization at specific genomic regions. Cell (2010) 8.79
Enhancing gene targeting with designed zinc finger nucleases. Science (2003) 8.42
Retroviral DNA integration: ASLV, HIV, and MLV show distinct target site preferences. PLoS Biol (2004) 8.27
A rapid and general assay for monitoring endogenous gene modification. Methods Mol Biol (2010) 6.77
Stimulation of homologous recombination through targeted cleavage by chimeric nucleases. Mol Cell Biol (2001) 6.68
Targeted gene knockout in mammalian cells by using engineered zinc-finger nucleases. Proc Natl Acad Sci U S A (2008) 6.59
Targeted gene addition into a specified location in the human genome using designed zinc finger nucleases. Proc Natl Acad Sci U S A (2007) 5.93
FokI dimerization is required for DNA cleavage. Proc Natl Acad Sci U S A (1998) 5.31
Requirements for double-strand cleavage by chimeric restriction enzymes with zinc finger DNA-recognition domains. Nucleic Acids Res (2000) 4.34
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. Genome Res (2010) 3.94
High-resolution insertion-site analysis by linear amplification-mediated PCR (LAM-PCR). Nat Methods (2007) 3.90
The discovery of zinc fingers and their applications in gene regulation and genome manipulation. Annu Rev Biochem (2010) 3.75
Role of the non-homologous DNA end joining pathway in the early steps of retroviral infection. EMBO J (2001) 3.60
Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy. J Clin Invest (2007) 3.28
Expanding or restricting the target site repertoire of zinc-finger nucleases: the inter-domain linker as a major determinant of target site selectivity. Mol Ther (2008) 3.02
FokI requires two specific DNA sites for cleavage. J Mol Biol (2001) 2.83
Rapid and efficient clathrin-mediated endocytosis revealed in genome-edited mammalian cells. Nat Cell Biol (2011) 2.79
Adeno-associated virus vectors integrate at chromosome breakage sites. Nat Genet (2004) 2.27
Genome-wide high-throughput integrome analyses by nrLAM-PCR and next-generation sequencing. Nat Protoc (2010) 2.24
Generation of a triple-gene knockout mammalian cell line using engineered zinc-finger nucleases. Biotechnol Bioeng (2010) 2.23
Comprehensive genomic access to vector integration in clinical gene therapy. Nat Med (2009) 2.19
Binding of two zinc finger nuclease monomers to two specific sites is required for effective double-strand DNA cleavage. Biochem Biophys Res Commun (2005) 2.15
Transient gene expression by nonintegrating lentiviral vectors. Mol Ther (2006) 1.89
Hepatocyte-targeted expression by integrase-defective lentiviral vectors induces antigen-specific tolerance in mice with low genotoxic risk. Hepatology (2011) 1.66
Capture of DNA sequences at double-strand breaks in mammalian chromosomes. Genetics (2001) 1.66
Targeted genome modifications using integrase-deficient lentiviral vectors. Mol Ther (2007) 1.60
Novel integrase-defective lentiviral episomal vectors for gene transfer. Hum Gene Ther (2004) 1.52
Frequent endonuclease cleavage at off-target locations in vivo. Mol Ther (2010) 1.52
Mutations in the human immunodeficiency virus type 1 integrase D,D(35)E motif do not eliminate provirus formation. J Virol (1998) 1.44
Non-homologous end-joining for repairing I-SceI-induced DNA double strand breaks in human cells. DNA Repair (Amst) (2007) 1.36
Promiscuous patching of broken chromosomes in mammalian cells with extrachromosomal DNA. Nucleic Acids Res (2001) 1.33
A TALE nuclease architecture for efficient genome editing. Nat Biotechnol (2010) 26.47
International network of cancer genome projects. Nature (2010) 20.35
Highly efficient endogenous human gene correction using designed zinc-finger nucleases. Nature (2005) 19.15
Genome editing with engineered zinc finger nucleases. Nat Rev Genet (2010) 18.88
An improved zinc-finger nuclease architecture for highly specific genome editing. Nat Biotechnol (2007) 17.80
Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases. Nat Biotechnol (2008) 14.75
Heritable targeted gene disruption in zebrafish using designed zinc-finger nucleases. Nat Biotechnol (2008) 14.20
Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases. Nat Biotechnol (2009) 12.40
Knockout rats via embryo microinjection of zinc-finger nucleases. Science (2009) 12.33
Genetic engineering of human pluripotent cells using TALE nucleases. Nat Biotechnol (2011) 11.76
Targeted genome editing across species using ZFNs and TALENs. Science (2011) 9.84
Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat Med (2006) 9.68
Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery. Nat Biotechnol (2007) 9.23
Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science (2009) 8.80
Distinct factors control histone variant H3.3 localization at specific genomic regions. Cell (2010) 8.79
A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N Engl J Med (2003) 8.59
Knockout rats generated by embryo microinjection of TALENs. Nat Biotechnol (2011) 8.45
Tie2 identifies a hematopoietic lineage of proangiogenic monocytes required for tumor vessel formation and a mesenchymal population of pericyte progenitors. Cancer Cell (2005) 7.90
Single-cell dissection of transcriptional heterogeneity in human colon tumors. Nat Biotechnol (2011) 7.84
Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV. N Engl J Med (2014) 7.64
Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. J Clin Invest (2008) 7.05
A rapid and general assay for monitoring endogenous gene modification. Methods Mol Biol (2010) 6.77
Whole-genome molecular haplotyping of single cells. Nat Biotechnol (2010) 6.77
Targeted gene knockout in mammalian cells by using engineered zinc-finger nucleases. Proc Natl Acad Sci U S A (2008) 6.59
Generation of isogenic pluripotent stem cells differing exclusively at two early onset Parkinson point mutations. Cell (2011) 6.56
Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo. Nat Biotechnol (2010) 6.55
Enhancing zinc-finger-nuclease activity with improved obligate heterodimeric architectures. Nat Methods (2010) 6.00
Targeted gene addition into a specified location in the human genome using designed zinc finger nucleases. Proc Natl Acad Sci U S A (2007) 5.93
Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. Science (2013) 5.64
Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration. Nat Biotechnol (2006) 5.58
Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. Lancet (2004) 5.43
Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state. Nat Biotechnol (2007) 5.36
Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science (2013) 5.34
Histone deimination antagonizes arginine methylation. Cell (2004) 5.19
Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells. Nature (2011) 4.92
Gene therapy: therapeutic gene causing lymphoma. Nature (2006) 4.89
In vivo genome editing restores haemostasis in a mouse model of haemophilia. Nature (2011) 4.81
Dissecting the genomic complexity underlying medulloblastoma. Nature (2012) 4.77
Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease. Nat Med (2010) 4.76
Precise genome modification in the crop species Zea mays using zinc-finger nucleases. Nature (2009) 4.71
Targeting exogenous genes to tumor angiogenesis by transplantation of genetically modified hematopoietic stem cells. Nat Med (2003) 4.02
Targeting the ANG2/TIE2 axis inhibits tumor growth and metastasis by impairing angiogenesis and disabling rebounds of proangiogenic myeloid cells. Cancer Cell (2011) 4.02
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. Genome Res (2010) 3.94
High-resolution insertion-site analysis by linear amplification-mediated PCR (LAM-PCR). Nat Methods (2007) 3.90
FcRgamma activation regulates inflammation-associated squamous carcinogenesis. Cancer Cell (2010) 3.80
Transient cold shock enhances zinc-finger nuclease-mediated gene disruption. Nat Methods (2010) 3.59
Murine leukemia induced by retroviral gene marking. Science (2002) 3.59
Stable knockdown of microRNA in vivo by lentiviral vectors. Nat Methods (2008) 3.55