Published in Nat Protoc on July 08, 2010
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Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. Science (2013) 5.64
Assessing the potential for AAV vector genotoxicity in a murine model. Blood (2010) 2.48
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Novel molecular and computational methods improve the accuracy of insertion site analysis in Sleeping Beauty-induced tumors. PLoS One (2011) 1.38
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Highly significant antiviral activity of HIV-1 LTR-specific tre-recombinase in humanized mice. PLoS Pathog (2013) 1.08
From skin biopsy to neurons through a pluripotent intermediate under Good Manufacturing Practice protocols. Stem Cells Transl Med (2011) 1.07
Lentiviral vector integration profiles differ in rodent postmitotic tissues. Mol Ther (2011) 1.06
Extensive methylation of promoter sequences silences lentiviral transgene expression during stem cell differentiation in vivo. Mol Ther (2012) 1.05
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Integration frequency and intermolecular recombination of rAAV vectors in non-human primate skeletal muscle and liver. Mol Ther (2012) 0.98
Challenges in vector and trial design using retroviral vectors for long-term gene correction in hematopoietic stem cell gene therapy. Mol Ther (2012) 0.98
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Therapeutic benefit of lentiviral-mediated neonatal intracerebral gene therapy in a mouse model of globoid cell leukodystrophy. Hum Mol Genet (2014) 0.94
The β-globin locus control region in combination with the EF1α short promoter allows enhanced lentiviral vector-mediated erythroid gene expression with conserved multilineage activity. Mol Ther (2012) 0.93
Retroviral vectors: post entry events and genomic alterations. Viruses (2011) 0.92
Retroviral gene therapy for X-linked chronic granulomatous disease: results from phase I/II trial. Mol Ther (2011) 0.91
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Preclinical evaluation of efficacy and safety of an improved lentiviral vector for the treatment of β-thalassemia and sickle cell disease. Curr Gene Ther (2015) 0.89
High-resolution analysis of the human T-cell receptor repertoire. Nat Commun (2015) 0.88
Cancer gene discovery: exploiting insertional mutagenesis. Mol Cancer Res (2013) 0.88
Engineered dendritic cells from cord blood and adult blood accelerate effector T cell immune reconstitution against HCMV. Mol Ther Methods Clin Dev (2015) 0.88
Fanconi anemia gene editing by the CRISPR/Cas9 system. Hum Gene Ther (2015) 0.87
Integration-deficient lentiviral vectors expressing codon-optimized R338L human FIX restore normal hemostasis in Hemophilia B mice. Mol Ther (2013) 0.87
Quantitative shearing linear amplification polymerase chain reaction: an improved method for quantifying lentiviral vector insertion sites in transplanted hematopoietic cell systems. Hum Gene Ther Methods (2015) 0.85
Presence of a trs-Like Motif Promotes Rep-Mediated Wild-Type Adeno-Associated Virus Type 2 Integration. J Virol (2015) 0.85
Integration profile and safety of an adenovirus hybrid-vector utilizing hyperactive sleeping beauty transposase for somatic integration. PLoS One (2013) 0.84
Stem cell gene therapy for fanconi anemia: report from the 1st international Fanconi anemia gene therapy working group meeting. Mol Ther (2011) 0.84
Transforming growth factor beta receptor 2 (TGFBR2) changes sialylation in the microsatellite unstable (MSI) Colorectal cancer cell line HCT116. PLoS One (2013) 0.84
Detecting DNA double-stranded breaks in mammalian genomes by linear amplification-mediated high-throughput genome-wide translocation sequencing. Nat Protoc (2016) 0.83
Integrase-deficient lentiviral vectors mediate efficient gene transfer to human vascular smooth muscle cells with minimal genotoxic risk. Hum Gene Ther (2012) 0.83
Transgenic expression of human glial cell line-derived neurotrophic factor from integration-deficient lentiviral vectors is neuroprotective in a rodent model of Parkinson's disease. Hum Gene Ther (2014) 0.82
Development of safer gene delivery systems to minimize the risk of insertional mutagenesis-related malignancies: a critical issue for the field of gene therapy. ISRN Oncol (2012) 0.82
Hybrid adeno-associated viral vectors utilizing transposase-mediated somatic integration for stable transgene expression in human cells. PLoS One (2013) 0.81
Lentiviral gene therapy using cellular promoters cures type 1 Gaucher disease in mice. Mol Ther (2015) 0.80
Ultrasound directs a transposase system for durable hepatic gene delivery in mice. Ultrasound Med Biol (2013) 0.80
Clonal Dominance With Retroviral Vector Insertions Near the ANGPT1 and ANGPT2 Genes in a Human Xenotransplant Mouse Model. Mol Ther Nucleic Acids (2014) 0.79
Identification of Hematopoietic Stem Cell Engraftment Genes in Gene Therapy Studies. J Stem Cell Res Ther (2013) 0.79
Linear amplification mediated PCR--localization of genetic elements and characterization of unknown flanking DNA. J Vis Exp (2014) 0.79
Integration site and clonal expansion in human chronic retroviral infection and gene therapy. Viruses (2014) 0.78
High-throughput monitoring of integration site clonality in preclinical and clinical gene therapy studies. Mol Ther Methods Clin Dev (2015) 0.77
Reactivating Fetal Hemoglobin Expression in Human Adult Erythroblasts Through BCL11A Knockdown Using Targeted Endonucleases. Mol Ther Nucleic Acids (2016) 0.76
Comparison Between Several Integrase-defective Lentiviral Vectors Reveals Increased Integration of an HIV Vector Bearing a D167H Mutant. Mol Ther Nucleic Acids (2014) 0.76
Pervasive supply of therapeutic lysosomal enzymes in the CNS of normal and Krabbe-affected non-human primates by intracerebral lentiviral gene therapy. EMBO Mol Med (2016) 0.75
An effective DNA vaccine platform for Middle East respiratory syndrome coronavirus. Ann Transl Med (2016) 0.75
Genome walking by next generation sequencing approaches. Biology (Basel) (2012) 0.75
Genetic Tagging During Human Mesoderm Differentiation Reveals Tripotent Lateral Plate Mesodermal Progenitors. Stem Cells (2016) 0.75
Vector integration and tumorigenesis. Hum Gene Ther (2014) 0.75
Monocyte-derived macrophages exhibit distinct and more restricted HIV-1 integration site repertoire than CD4(+) T cells. Sci Rep (2016) 0.75
Genome-wide Specificity of Highly Efficient TALENs and CRISPR/Cas9 for T Cell Receptor Modification. Mol Ther Methods Clin Dev (2017) 0.75
Genetic barcode sequencing for screening altered population dynamics of hematopoietic stem cells transduced with lentivirus. Mol Ther Methods Clin Dev (2014) 0.75
TRE5-A retrotransposition profiling reveals putative RNA polymerase III transcription complex binding sites on the Dictyostelium extrachromosomal rDNA element. PLoS One (2017) 0.75
VISMapper: ultra-fast exhaustive cartography of viral insertion sites for gene therapy. BMC Bioinformatics (2017) 0.75
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LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science (2003) 19.77
In vivo footprinting of a muscle specific enhancer by ligation mediated PCR. Science (1989) 10.87
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
Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science (2009) 8.80
A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N Engl J Med (2003) 8.59
Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. J Clin Invest (2008) 7.05
Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science (2002) 6.46
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
A pyrosequencing-tailored nucleotide barcode design unveils opportunities for large-scale sample multiplexing. Nucleic Acids Res (2007) 5.33
From micrograms to picograms: quantitative PCR reduces the material demands of high-throughput sequencing. Nucleic Acids Res (2007) 4.26
High-resolution insertion-site analysis by linear amplification-mediated PCR (LAM-PCR). Nat Methods (2007) 3.90
The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy. J Clin Invest (2009) 3.34
Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy. J Clin Invest (2007) 3.28
Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity. Blood (2006) 3.07
Novel use of polymerase chain reaction to amplify cellular DNA adjacent to an integrated provirus. J Virol (1989) 2.92
Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo. J Clin Invest (2007) 2.80
DNA bar coding and pyrosequencing to analyze adverse events in therapeutic gene transfer. Nucleic Acids Res (2008) 2.64
Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy. J Clin Invest (2007) 2.63
Polyclonal long-term repopulating stem cell clones in a primate model. Blood (2002) 2.53
Comprehensive genomic access to vector integration in clinical gene therapy. Nat Med (2009) 2.19
Short PCR product yields improved by lower denaturation temperatures. Nucleic Acids Res (1991) 1.58
Adeno-associated virus vector genomes persist as episomal chromatin in primate muscle. J Virol (2008) 1.44
Multiarm high-throughput integration site detection: limitations of LAM-PCR technology and optimization for clonal analysis. Stem Cells Dev (2007) 1.32
Genotoxicity in gene therapy: an account of vector integration and designer nucleases. Curr Opin Mol Ther (2008) 1.04
Direct PCR of intact bacteria (colony PCR). Curr Protoc Microbiol (2008) 0.97
A second generation human haplotype map of over 3.1 million SNPs. Nature (2007) 85.39
Scalable molecular dynamics with NAMD. J Comput Chem (2005) 59.49
The diploid genome sequence of an Asian individual. Nature (2008) 46.29
Distinct and predictive chromatin signatures of transcriptional promoters and enhancers in the human genome. Nat Genet (2007) 32.41
International network of cancer genome projects. Nature (2010) 20.35
Genome-wide detection and characterization of positive selection in human populations. Nature (2007) 17.27
Genome-wide association scan in women with systemic lupus erythematosus identifies susceptibility variants in ITGAM, PXK, KIAA1542 and other loci. Nat Genet (2008) 12.51
piggyBac transposition reprograms fibroblasts to induced pluripotent stem cells. Nature (2009) 11.27
Sequencing of 50 human exomes reveals adaptation to high altitude. Science (2010) 11.27
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
Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science (2009) 8.80
Distinct epigenomic landscapes of pluripotent and lineage-committed human cells. Cell Stem Cell (2010) 8.74
A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N Engl J Med (2003) 8.59
A two-gene expression ratio predicts clinical outcome in breast cancer patients treated with tamoxifen. Cancer Cell (2004) 8.06
Automatic atom type and bond type perception in molecular mechanical calculations. J Mol Graph Model (2006) 7.93
Targeted bisulfite sequencing reveals changes in DNA methylation associated with nuclear reprogramming. Nat Biotechnol (2009) 7.59
Oncogenic Kras maintains pancreatic tumors through regulation of anabolic glucose metabolism. Cell (2012) 7.36
Acute renal failure and sepsis. N Engl J Med (2004) 7.18
The MicroArray Quality Control (MAQC)-II study of common practices for the development and validation of microarray-based predictive models. Nat Biotechnol (2010) 7.08
Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. J Clin Invest (2008) 7.05
High-performance neuroprosthetic control by an individual with tetraplegia. Lancet (2012) 6.66
An unbiased genome-wide analysis of zinc-finger nuclease specificity. Nat Biotechnol (2011) 6.44
The dog genome: survey sequencing and comparative analysis. Science (2003) 5.84
Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. Science (2013) 5.64
New loci associated with kidney function and chronic kidney disease. Nat Genet (2010) 5.58
Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration. Nat Biotechnol (2006) 5.58
Antitumor activity of rapamycin in a Phase I trial for patients with recurrent PTEN-deficient glioblastoma. PLoS Med (2008) 5.54
The draft genome of the transgenic tropical fruit tree papaya (Carica papaya Linnaeus). Nature (2008) 5.54
Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. Lancet (2004) 5.43
Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science (2013) 5.34
Predictive value for the Chinese population of the Framingham CHD risk assessment tool compared with the Chinese Multi-Provincial Cohort Study. JAMA (2004) 5.34
The oyster genome reveals stress adaptation and complexity of shell formation. Nature (2012) 5.30
De novo mutations in histone-modifying genes in congenital heart disease. Nature (2013) 5.15
Evidence for HIV-associated B cell exhaustion in a dysfunctional memory B cell compartment in HIV-infected viremic individuals. J Exp Med (2008) 5.14
MicroRNA-34b and MicroRNA-34c are targets of p53 and cooperate in control of cell proliferation and adhesion-independent growth. Cancer Res (2007) 4.95
Gene therapy: therapeutic gene causing lymphoma. Nature (2006) 4.89
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
Hepatocyte growth factor induces gefitinib resistance of lung adenocarcinoma with epidermal growth factor receptor-activating mutations. Cancer Res (2008) 4.30
EpCAM and alpha-fetoprotein expression defines novel prognostic subtypes of hepatocellular carcinoma. Cancer Res (2008) 4.17
Probable limited person-to-person transmission of highly pathogenic avian influenza A (H5N1) virus in China. Lancet (2008) 4.17
High-resolution insertion-site analysis by linear amplification-mediated PCR (LAM-PCR). Nat Methods (2007) 3.90
Pelvic Organ Support Study (POSST): the distribution, clinical definition, and epidemiologic condition of pelvic organ support defects. Am J Obstet Gynecol (2005) 3.85
Association between adverse clinical outcome in human disease caused by novel influenza A H7N9 virus and sustained viral shedding and emergence of antiviral resistance. Lancet (2013) 3.83
Epigenomic analysis of multilineage differentiation of human embryonic stem cells. Cell (2013) 3.81
Epidemiological transition of stroke in China: twenty-one-year observational study from the Sino-MONICA-Beijing Project. Stroke (2008) 3.81
Natural selection on EPAS1 (HIF2alpha) associated with low hemoglobin concentration in Tibetan highlanders. Proc Natl Acad Sci U S A (2010) 3.78
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Discovery and annotation of functional chromatin signatures in the human genome. PLoS Comput Biol (2009) 3.61
Murine leukemia induced by retroviral gene marking. Science (2002) 3.59
Residential proximity to naturally occurring asbestos and mesothelioma risk in California. Am J Respir Crit Care Med (2005) 3.55
Clinical application of massively parallel sequencing-based prenatal noninvasive fetal trisomy test for trisomies 21 and 18 in 11,105 pregnancies with mixed risk factors. Prenat Diagn (2012) 3.54
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Distinct genomic integration of MLV and SIV vectors in primate hematopoietic stem and progenitor cells. PLoS Biol (2004) 3.48
ChromaSig: a probabilistic approach to finding common chromatin signatures in the human genome. PLoS Comput Biol (2008) 3.46
The honey bee epigenomes: differential methylation of brain DNA in queens and workers. PLoS Biol (2010) 3.42
A proprotein convertase subtilisin/kexin type 9 neutralizing antibody reduces serum cholesterol in mice and nonhuman primates. Proc Natl Acad Sci U S A (2009) 3.39
Characteristics associated with differences in survival among black and white women with breast cancer. JAMA (2013) 3.35
The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy. J Clin Invest (2009) 3.34
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Allele-specific methylation is prevalent and is contributed by CpG-SNPs in the human genome. Genome Res (2010) 3.17
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A recalibrated molecular clock and independent origins for the cholera pandemic clones. PLoS One (2008) 3.16
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Stem-cell gene therapy for the Wiskott-Aldrich syndrome. N Engl J Med (2010) 3.09
Quantum-sized carbon dots for bright and colorful photoluminescence. J Am Chem Soc (2006) 3.08
Adverse metabolic consequences in humans of prolonged sleep restriction combined with circadian disruption. Sci Transl Med (2012) 3.08
Integrative genomic analyses reveal an androgen-driven somatic alteration landscape in early-onset prostate cancer. Cancer Cell (2013) 3.08