Published in Mol Ther on January 12, 2010
In vivo genome editing using a high-efficiency TALEN system. Nature (2012) 7.11
Generation of isogenic pluripotent stem cells differing exclusively at two early onset Parkinson point mutations. Cell (2011) 6.56
High-frequency genome editing using ssDNA oligonucleotides with zinc-finger nucleases. Nat Methods (2011) 5.68
Zinc-finger nuclease-driven targeted integration into mammalian genomes using donors with limited chromosomal homology. Nucleic Acids Res (2010) 2.61
Obligate ligation-gated recombination (ObLiGaRe): custom-designed nuclease-mediated targeted integration through nonhomologous end joining. Genome Res (2012) 2.12
Advances in targeted genome editing. Curr Opin Chem Biol (2012) 1.97
Donor DNA Utilization During Gene Targeting with Zinc-Finger Nucleases. G3 (Bethesda) (2013) 1.92
Autonomous zinc-finger nuclease pairs for targeted chromosomal deletion. Nucleic Acids Res (2010) 1.92
Inducible cassette exchange: a rapid and efficient system enabling conditional gene expression in embryonic stem and primary cells. Stem Cells (2011) 1.59
ZFN-site searches genomes for zinc finger nuclease target sites and off-target sites. BMC Bioinformatics (2011) 1.16
Efficient homologous recombination-mediated genome engineering in zebrafish using TALE nucleases. Development (2014) 1.16
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
Hematopoietic stem cell engineering at a crossroads. Blood (2011) 1.10
Efficient and accurate homologous recombination in hESCs and hiPSCs using helper-dependent adenoviral vectors. Mol Ther (2011) 1.06
Transcription activator like effector (TALE)-directed piggyBac transposition in human cells. Nucleic Acids Res (2013) 1.01
ssODN-mediated knock-in with CRISPR-Cas for large genomic regions in zygotes. Nat Commun (2016) 0.99
New and TALENted genome engineering toolbox. Circ Res (2013) 0.96
High efficiency In Vivo genome engineering with a simplified 15-RVD GoldyTALEN design. PLoS One (2013) 0.95
Efficient bi-allelic gene knockout and site-specific knock-in mediated by TALENs in pigs. Sci Rep (2014) 0.94
Targeted gene therapies: tools, applications, optimization. Crit Rev Biochem Mol Biol (2012) 0.91
Repeatable construction method for engineered zinc finger nuclease based on overlap extension PCR and TA-cloning. PLoS One (2013) 0.90
Analysis of illegitimate genomic integration mediated by zinc-finger nucleases: implications for specificity of targeted gene correction. BMC Mol Biol (2010) 0.89
Genome editing revolutionize the creation of genetically modified pigs for modeling human diseases. Hum Genet (2016) 0.88
Oligo/polynucleotide-based gene modification: strategies and therapeutic potential. Oligonucleotides (2011) 0.87
Genome Editing in Human Pluripotent Stem Cells: Approaches, Pitfalls, and Solutions. Cell Stem Cell (2016) 0.87
An update on targeted gene repair in mammalian cells: methods and mechanisms. J Biomed Sci (2011) 0.84
Light-activated gene editing with a photocaged zinc-finger nuclease. Angew Chem Int Ed Engl (2011) 0.83
Rosa26 locus supports tissue-specific promoter driving transgene expression specifically in pig. PLoS One (2014) 0.82
RecA-independent single-stranded DNA oligonucleotide-mediated mutagenesis. F1000 Biol Rep (2010) 0.82
Analysis of SOX2-expressing cell populations derived from human pluripotent stem cells. Stem Cell Reports (2013) 0.81
BTK gene targeting by homologous recombination using a helper-dependent adenovirus/adeno-associated virus hybrid vector. Gene Ther (2015) 0.80
Emerging therapies for mitochondrial disorders. Brain (2016) 0.80
Electrospun fiber membranes enable proliferation of genetically modified cells. Int J Nanomedicine (2013) 0.79
Stimulation of oligonucleotide-directed gene correction by Redβ expression and MSH2 depletion in human HT1080 cells. Mol Cells (2014) 0.76
Virus-mediated Genetic Surgery: Homologous Recombination With a Little "Helper" From My Friends. Mol Ther Nucleic Acids (2012) 0.75
Targeting Human α-Lactalbumin Gene Insertion into the Goat β-Lactoglobulin Locus by TALEN-Mediated Homologous Recombination. PLoS One (2016) 0.75
LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science (2003) 19.77
An improved zinc-finger nuclease architecture for highly specific genome editing. Nat Biotechnol (2007) 17.80
Generation of mouse induced pluripotent stem cells without viral vectors. Science (2008) 15.25
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 therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science (2000) 10.40
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
Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J Clin Invest (2008) 9.42
Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery. Nat Biotechnol (2007) 9.23
Structure-based redesign of the dimerization interface reduces the toxicity of zinc-finger nucleases. Nat Biotechnol (2007) 8.88
Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. J Clin Invest (2008) 7.05
Stimulation of homologous recombination through targeted cleavage by chimeric nucleases. Mol Cell Biol (2001) 6.68
Zinc-finger nucleases: the next generation emerges. Mol Ther (2008) 5.69
Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. Lancet (2004) 5.43
Requirements for double-strand cleavage by chimeric restriction enzymes with zinc finger DNA-recognition domains. Nucleic Acids Res (2000) 4.34
Zinc Finger Tools: custom DNA-binding domains for transcription factors and nucleases. Nucleic Acids Res (2006) 3.95
DNA-binding specificity is a major determinant of the activity and toxicity of zinc-finger nucleases. Mol Ther (2007) 3.94
Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy. J Clin Invest (2007) 3.28
Custom zinc-finger nucleases for use in human cells. Mol Ther (2005) 3.04
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
Oligomerized pool engineering (OPEN): an 'open-source' protocol for making customized zinc-finger arrays. Nat Protoc (2009) 3.01
Reprogramming cell fates: reconciling rarity with robustness. Bioessays (2009) 2.38
Design, activity, and structure of a highly specific artificial endonuclease. Mol Cell (2002) 2.25
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
Efficient targeting of a SCID gene by an engineered single-chain homing endonuclease. Nucleic Acids Res (2009) 2.14
Efficient gene targeting mediated by adeno-associated virus and DNA double-strand breaks. Mol Cell Biol (2003) 2.01
Analysis of lentiviral vector integration in HIV+ study subjects receiving autologous infusions of gene modified CD4+ T cells. Mol Ther (2009) 1.97
Extensive genomic copy number variation in embryonic stem cells. Proc Natl Acad Sci U S A (2008) 1.91
Leukemias following retroviral transfer of multidrug resistance 1 (MDR1) are driven by combinatorial insertional mutagenesis. Blood (2005) 1.82
Chimeric restriction enzymes: what is next? Biol Chem (1999) 1.76
Targeted genome modifications using integrase-deficient lentiviral vectors. Mol Ther (2007) 1.60
Mutations altering the cleavage specificity of a homing endonuclease. Nucleic Acids Res (2002) 1.60
Targeted gene correction by small single-stranded oligonucleotides in mammalian cells. Gene Ther (2001) 1.46
Splase: a new class IIS zinc-finger restriction endonuclease with specificity for Sp1 binding sites. J Protein Chem (1996) 1.36
Genomic sequence correction by single-stranded DNA oligonucleotides: role of DNA synthesis and chemical modifications of the oligonucleotide ends. J Gene Med (2005) 1.29
Targeted gene modification in mismatch-repair-deficient embryonic stem cells by single-stranded DNA oligonucleotides. Nucleic Acids Res (2003) 1.28
Regulation of targeted gene repair by intrinsic cellular processes. Bioessays (2009) 1.23
Sequence-specific modification of mitochondrial DNA using a chimeric zinc finger methylase. Proc Natl Acad Sci U S A (2006) 1.22
Targeted chromosomal gene modification in human cells by single-stranded oligodeoxynucleotides in the presence of a DNA double-strand break. Mol Ther (2006) 1.16
Karyotype analysis of the euploid cell population of a mouse embryonic stem cell line revealed a high incidence of chromosome abnormalities that varied during culture. Cytogenet Genome Res (2008) 1.08
Targeted gene repair in mammalian cells using chimeric RNA/DNA oligonucleotides and modified single-stranded vectors. Sci STKE (2001) 0.91
Unmodified oligodeoxynucleotides require single-strandedness to induce targeted repair of a chromosomal EGFP gene. J Gene Med (2004) 0.90
Generation and functional analysis of zinc finger nucleases. Methods Mol Biol (2008) 0.89
Targeted gene correction of hprt mutations by 45 base single-stranded oligonucleotides. Biochem Biophys Res Commun (2002) 0.88
Rapid "open-source" engineering of customized zinc-finger nucleases for highly efficient gene modification. Mol Cell (2008) 11.24
Structure-based redesign of the dimerization interface reduces the toxicity of zinc-finger nucleases. Nat Biotechnol (2007) 8.88
A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity. Nucleic Acids Res (2011) 7.93
Unexpected failure rates for modular assembly of engineered zinc fingers. Nat Methods (2008) 6.51
Mechanism and regulation of human non-homologous DNA end-joining. Nat Rev Mol Cell Biol (2003) 6.49
Hairpin opening and overhang processing by an Artemis/DNA-dependent protein kinase complex in nonhomologous end joining and V(D)J recombination. Cell (2002) 6.34
DNA-binding specificity is a major determinant of the activity and toxicity of zinc-finger nucleases. Mol Ther (2007) 3.94
Regulated expression of nuclear receptor RORγt confers distinct functional fates to NK cell receptor-expressing RORγt(+) innate lymphocytes. Immunity (2010) 3.59
Custom zinc-finger nucleases for use in human cells. Mol Ther (2005) 3.04
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
A biochemically defined system for mammalian nonhomologous DNA end joining. Mol Cell (2004) 3.01
Engineered zinc finger nickases induce homology-directed repair with reduced mutagenic effects. Nucleic Acids Res (2012) 2.26
Differential integrity of TALE nuclease genes following adenoviral and lentiviral vector gene transfer into human cells. Nucleic Acids Res (2012) 2.14
Efficient gene targeting mediated by adeno-associated virus and DNA double-strand breaks. Mol Cell Biol (2003) 2.01
TALE nucleases: tailored genome engineering made easy. Curr Opin Biotechnol (2012) 1.96
The mechanism of vertebrate nonhomologous DNA end joining and its role in V(D)J recombination. DNA Repair (Amst) (2004) 1.93
Autonomous zinc-finger nuclease pairs for targeted chromosomal deletion. Nucleic Acids Res (2010) 1.92
Autoimmunity in Wiskott-Aldrich syndrome: risk factors, clinical features, and outcome in a single-center cohort of 55 patients. Pediatrics (2003) 1.91
Circulating CD21low B cells in common variable immunodeficiency resemble tissue homing, innate-like B cells. Proc Natl Acad Sci U S A (2009) 1.91
The DNA-dependent protein kinase catalytic subunit phosphorylation sites in human Artemis. J Biol Chem (2005) 1.84
RNA guides genome engineering. Nat Biotechnol (2013) 1.84
Mutations in the von Hippel-Lindau (VHL) tumor suppressor gene and VHL-haplotype analysis in patients with presumable congenital erythrocytosis. Haematologica (2005) 1.83
The Artemis:DNA-PKcs endonuclease cleaves DNA loops, flaps, and gaps. DNA Repair (Amst) (2005) 1.81
An immunodeficiency disease with RAG mutations and granulomas. N Engl J Med (2008) 1.71
SCID patients with ARTEMIS vs RAG deficiencies following HCT: increased risk of late toxicity in ARTEMIS-deficient SCID. Blood (2013) 1.68
Length-dependent binding of human XLF to DNA and stimulation of XRCC4.DNA ligase IV activity. J Biol Chem (2007) 1.65
Dihydrofolate reductase deficiency due to a homozygous DHFR mutation causes megaloblastic anemia and cerebral folate deficiency leading to severe neurologic disease. Am J Hum Genet (2011) 1.63
Targeted genome modifications using integrase-deficient lentiviral vectors. Mol Ther (2007) 1.60
Zinc-finger nuclease based genome surgery: it's all about specificity. Curr Gene Ther (2011) 1.56
Single-stranded DNA ligation and XLF-stimulated incompatible DNA end ligation by the XRCC4-DNA ligase IV complex: influence of terminal DNA sequence. Nucleic Acids Res (2007) 1.56
Functional and biochemical dissection of the structure-specific nuclease ARTEMIS. EMBO J (2004) 1.54
A variant of SCID with specific immune responses and predominance of gamma delta T cells. J Clin Invest (2005) 1.54
Targeted genome editing in pluripotent stem cells using zinc-finger nucleases. Methods (2010) 1.48
Omenn syndrome due to ARTEMIS mutations. Blood (2005) 1.47
Lethal hemophagocytic lymphohistiocytosis in Hermansky-Pudlak syndrome type II. Blood (2006) 1.42
The embryonic lethality in DNA ligase IV-deficient mice is rescued by deletion of Ku: implications for unifying the heterogeneous phenotypes of NHEJ mutants. DNA Repair (Amst) (2002) 1.41
Results of intracoronary stem cell therapy after acute myocardial infarction. Am J Cardiol (2010) 1.40
Clinical and immunological manifestations of patients with atypical severe combined immunodeficiency. Clin Immunol (2011) 1.38
Deficiency of innate and acquired immunity caused by an IKBKB mutation. N Engl J Med (2013) 1.37
ORAI1-mediated calcium influx is required for human cytotoxic lymphocyte degranulation and target cell lysis. Proc Natl Acad Sci U S A (2011) 1.35
Physical incorporation of a single-stranded oligodeoxynucleotide during targeted repair of a human chromosomal locus. J Gene Med (2006) 1.35
Reticular dysgenesis (aleukocytosis) is caused by mutations in the gene encoding mitochondrial adenylate kinase 2. Nat Genet (2008) 1.34
Antiviral and regulatory T cell immunity in a patient with stromal interaction molecule 1 deficiency. J Immunol (2011) 1.32
MicroRNA-221 overexpression accelerates hepatocyte proliferation during liver regeneration. Hepatology (2013) 1.32
A severe form of human combined immunodeficiency due to mutations in DNA ligase IV. J Immunol (2006) 1.31
High-resolution HLA matching in hematopoietic stem cell transplantation: a retrospective collaborative analysis. Blood (2013) 1.30
Breaking chemoresistance and radioresistance with [213Bi]anti-CD45 antibodies in leukemia cells. Cancer Res (2007) 1.29
Repair of double-strand DNA breaks by the human nonhomologous DNA end joining pathway: the iterative processing model. Cell Cycle (2005) 1.28
DNA-PKcs dependence of Artemis endonucleolytic activity, differences between hairpins and 5' or 3' overhangs. J Biol Chem (2006) 1.28
Inactivation of hepatitis B virus replication in cultured cells and in vivo with engineered transcription activator-like effector nucleases. Mol Ther (2013) 1.26
Quantification of zinc finger nuclease-associated toxicity. Methods Mol Biol (2010) 1.24
Adding fingers to an engineered zinc finger nuclease can reduce activity. Biochemistry (2011) 1.17
Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases. Mol Ther (2013) 1.16
Targeted chromosomal gene modification in human cells by single-stranded oligodeoxynucleotides in the presence of a DNA double-strand break. Mol Ther (2006) 1.16
A novel single point mutation of the LYST gene in two siblings with different phenotypic features of Chediak Higashi syndrome. Pediatr Blood Cancer (2011) 1.16
Congenital dyserythropoietic anemia type I (CDA I): molecular genetics, clinical appearance, and prognosis based on long-term observation. Blood (2005) 1.16
Atypical familial hemophagocytic lymphohistiocytosis due to mutations in UNC13D and STXBP2 overlaps with primary immunodeficiency diseases. Haematologica (2010) 1.15
A biochemically defined system for coding joint formation in V(D)J recombination. Mol Cell (2008) 1.13
Peptide nanofibrils boost retroviral gene transfer and provide a rapid means for concentrating viruses. Nat Nanotechnol (2013) 1.12
Subtle differences in CTL cytotoxicity determine susceptibility to hemophagocytic lymphohistiocytosis in mice and humans with Chediak-Higashi syndrome. Blood (2011) 1.12
Integration preferences of wildtype AAV-2 for consensus rep-binding sites at numerous loci in the human genome. PLoS Pathog (2010) 1.12
Zinc-finger nucleases for somatic gene therapy: the next frontier. Hum Gene Ther (2011) 1.11
DNA-PKcs regulates a single-stranded DNA endonuclease activity of Artemis. DNA Repair (Amst) (2010) 1.07
The morphological diagnosis of congenital dyserythropoietic anemia: results of a quantitative analysis of peripheral blood and bone marrow cells. Haematologica (2010) 1.06
Patients with adenosine deaminase deficiency surviving after hematopoietic stem cell transplantation are at high risk of CNS complications. Blood (2006) 1.06
Genotoxicity in gene therapy: an account of vector integration and designer nucleases. Curr Opin Mol Ther (2008) 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
Retroviral WASP gene transfer into human hematopoietic stem cells reconstitutes the actin cytoskeleton in myeloid progeny cells differentiated in vitro. Exp Hematol (2006) 1.03
Clinical and immunologic consequences of a somatic reversion in a patient with X-linked severe combined immunodeficiency. Blood (2008) 1.02
MicroRNA-221 regulates FAS-induced fulminant liver failure. Hepatology (2011) 1.01
Ultramicroscopy reveals axonal transport impairments in cortical motor neurons at prion disease. Biophys J (2009) 1.00
Omenn syndrome: a lack of tolerance on the background of deficient lymphocyte development and maturation. Curr Opin Rheumatol (2006) 0.99
Retargeting sleeping beauty transposon insertions by engineered zinc finger DNA-binding domains. Mol Ther (2012) 0.97
Two siblings with lethal pneumococcal meningitis in a family with a mutation in Interleukin-1 receptor-associated kinase 4. J Pediatr (2004) 0.97
Autoimmunity, autoinflammation and lymphoma in combined immunodeficiency (CID). Autoimmun Rev (2010) 0.97
The risk of hemophagocytic lymphohistiocytosis in Hermansky-Pudlak syndrome type 2. Blood (2013) 0.97
Growth differentiation factor 15 in patients with congenital dyserythropoietic anaemia (CDA) type II. J Mol Med (Berl) (2011) 0.95
Sequential decisions on FAS sequencing guided by biomarkers in patients with lymphoproliferation and autoimmune cytopenia. Haematologica (2013) 0.95
Telomere elongation and clinical response to androgen treatment in a patient with aplastic anemia and a heterozygous hTERT gene mutation. Ann Hematol (2012) 0.94
Frequency of congenital dyserythropoietic anemias in Europe. Eur J Haematol (2010) 0.94
Fate of recombinant adeno-associated viral vector genomes during DNA double-strand break-induced gene targeting in human cells. Hum Gene Ther (2010) 0.94
Extent to which hairpin opening by the Artemis:DNA-PKcs complex can contribute to junctional diversity in V(D)J recombination. Nucleic Acids Res (2007) 0.93
Retargeting transposon insertions by the adeno-associated virus Rep protein. Nucleic Acids Res (2012) 0.92
A novel zinc-finger nuclease platform with a sequence-specific cleavage module. Nucleic Acids Res (2011) 0.92
Generation and genetic modification of induced pluripotent stem cells. Expert Opin Biol Ther (2010) 0.91
Matricellular signaling molecule CCN1 attenuates experimental autoimmune myocarditis by acting as a novel immune cell migration modulator. Circulation (2010) 0.91
The most frequent DCLRE1C (ARTEMIS) mutations are based on homologous recombination events. Hum Mutat (2010) 0.90
Primary necrotizing lymphocytic central nervous system vasculitis due to perforin deficiency in a four-year-old girl. Arthritis Rheum (2007) 0.90
Unmodified oligodeoxynucleotides require single-strandedness to induce targeted repair of a chromosomal EGFP gene. J Gene Med (2004) 0.90
Molecular genetic analyses in familial and sporadic congenital primary erythrocytosis. Haematologica (2007) 0.90
DNA-ligase IV and DNA-protein kinase play a critical role in deficient caspases activation in apoptosis-resistant cancer cells by using doxorubicin. Mol Biol Cell (2008) 0.89
Efficient transient genetic labeling of human CD34+ progenitor cells for in vivo application. Regen Med (2006) 0.87
The nontoxic cell cycle modulator indirubin augments transduction of adeno-associated viral vectors and zinc-finger nuclease-mediated gene targeting. Hum Gene Ther (2012) 0.86
Selection-independent generation of gene knockout mouse embryonic stem cells using zinc-finger nucleases. PLoS One (2011) 0.85
Histone deacetylase inhibition rescues gene knockout levels achieved with integrase-defective lentiviral vectors encoding zinc-finger nucleases. Hum Gene Ther Methods (2013) 0.84