Published in Nat Med on January 24, 2010
Gene Therapy for Chronic Granulomatous Disease (CGD) | NCT00564759
Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. Science (2013) 5.64
Oxidase-deficient neutrophils from X-linked chronic granulomatous disease iPS cells: functional correction by zinc finger nuclease-mediated safe harbor targeting. Blood (2011) 3.21
Stem-cell gene therapy for the Wiskott-Aldrich syndrome. N Engl J Med (2010) 3.09
Unraveling the molecular pathophysiology of myelodysplastic syndromes. J Clin Oncol (2011) 2.69
Assessing the potential for AAV vector genotoxicity in a murine model. Blood (2010) 2.48
Aberrant DNA hypermethylation signature in acute myeloid leukemia directed by EVI1. Blood (2010) 2.15
Ex vivo gene transfer and correction for cell-based therapies. Nat Rev Genet (2011) 2.13
Gene therapy returns to centre stage. Nature (2015) 1.95
NY-ESO-1-specific TCR-engineered T cells mediate sustained antigen-specific antitumor effects in myeloma. Nat Med (2015) 1.92
Allogeneic hematopoietic stem cell transplantation for sickle cell disease: the time is now. Blood (2011) 1.90
Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans. Blood (2012) 1.85
Genome editing of human embryonic stem cells and induced pluripotent stem cells with zinc finger nucleases for cellular imaging. Circ Res (2012) 1.84
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Lentiviral vector design and imaging approaches to visualize the early stages of cellular reprogramming. Mol Ther (2011) 1.69
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Adoptive immunotherapy for cancer or viruses. Annu Rev Immunol (2014) 1.61
Genomic discovery of potent chromatin insulators for human gene therapy. Nat Biotechnol (2015) 1.56
Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012. Hum Gene Ther (2013) 1.54
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Clinical efficacy of gene-modified stem cells in adenosine deaminase-deficient immunodeficiency. J Clin Invest (2017) 1.39
Chronic granulomatous disease: lessons from a rare disorder. Biol Blood Marrow Transplant (2011) 1.38
Evi1 is essential for hematopoietic stem cell self-renewal, and its expression marks hematopoietic cells with long-term multilineage repopulating activity. J Exp Med (2011) 1.36
Retroviral integrations in gene therapy trials. Mol Ther (2012) 1.34
Bromo- and extraterminal domain chromatin regulators serve as cofactors for murine leukemia virus integration. J Virol (2013) 1.34
Gene therapy of chronic granulomatous disease: the engraftment dilemma. Mol Ther (2010) 1.34
Extended survival of glioblastoma patients after chemoprotective HSC gene therapy. Sci Transl Med (2012) 1.27
A remote GATA2 hematopoietic enhancer drives leukemogenesis in inv(3)(q21;q26) by activating EVI1 expression. Cancer Cell (2014) 1.27
Methylation and silencing of miRNA-124 by EVI1 and self-renewal exhaustion of hematopoietic stem cells in murine myelodysplastic syndrome. Proc Natl Acad Sci U S A (2010) 1.25
Gene therapy for PIDs: progress, pitfalls and prospects. Gene (2013) 1.22
Bioinformatic clonality analysis of next-generation sequencing-derived viral vector integration sites. Hum Gene Ther Methods (2012) 1.21
Lentiviral vector integration in the human genome induces alternative splicing and generates aberrant transcripts. J Clin Invest (2012) 1.19
Preclinical safety and efficacy of human CD34(+) cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich syndrome. Mol Ther (2012) 1.18
Current advances in retroviral gene therapy. Curr Gene Ther (2011) 1.13
Two decades of clinical gene therapy--success is finally mounting. Discov Med (2010) 1.13
Derivation and functional analysis of patient-specific induced pluripotent stem cells as an in vitro model of chronic granulomatous disease. Stem Cells (2012) 1.11
Biochemical correction of X-CGD by a novel chimeric promoter regulating high levels of transgene expression in myeloid cells. Mol Ther (2010) 1.11
Hematopoietic stem cell engineering at a crossroads. Blood (2011) 1.10
Whole transcriptome characterization of aberrant splicing events induced by lentiviral vector integrations. J Clin Invest (2012) 1.10
Gene therapy on the move. EMBO Mol Med (2013) 1.10
Analyzing the number of common integration sites of viral vectors--new methods and computer programs. PLoS One (2011) 1.10
Targeted gene addition in human epithelial stem cells by zinc-finger nuclease-mediated homologous recombination. Mol Ther (2013) 1.10
Insertional oncogenesis by non-acute retroviruses: implications for gene therapy. Viruses (2011) 1.09
Genotoxicity of retroviral hematopoietic stem cell gene therapy. Expert Opin Biol Ther (2011) 1.08
High-definition mapping of retroviral integration sites defines the fate of allogeneic T cells after donor lymphocyte infusion. PLoS One (2010) 1.07
Extensive methylation of promoter sequences silences lentiviral transgene expression during stem cell differentiation in vivo. Mol Ther (2012) 1.05
Stem cell gene therapy: the risks of insertional mutagenesis and approaches to minimize genotoxicity. Front Med (2011) 1.05
Insertion sites in engrafted cells cluster within a limited repertoire of genomic areas after gammaretroviral vector gene therapy. Mol Ther (2011) 1.04
Advantages and applications of CAR-expressing natural killer cells. Front Pharmacol (2015) 1.03
Gene therapy for primary immunodeficiencies. Hum Gene Ther (2012) 1.03
Targeted gene addition in human CD34(+) hematopoietic cells for correction of X-linked chronic granulomatous disease. Nat Biotechnol (2016) 1.03
Dynamics of HSPC repopulation in nonhuman primates revealed by a decade-long clonal-tracking study. Cell Stem Cell (2014) 1.02
Evaluating risks of insertional mutagenesis by DNA transposons in gene therapy. Transl Res (2013) 1.01
Primary Immune Deficiency Treatment Consortium (PIDTC) report. J Allergy Clin Immunol (2013) 1.01
Lentiviral vector induced insertional haploinsufficiency of Ebf1 causes murine leukemia. Mol Ther (2012) 1.01
Biosafety features of lentiviral vectors. Hum Gene Ther (2013) 1.01
Molecular mechanisms of retroviral integration site selection. Nucleic Acids Res (2014) 1.01
Lentiviral vector gene therapy: effective and safe? Mol Ther (2010) 0.99
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
Distribution of lentiviral vector integration sites in mice following therapeutic gene transfer to treat β-thalassemia. Mol Ther (2011) 0.98
Alpharetroviral self-inactivating vectors: long-term transgene expression in murine hematopoietic cells and low genotoxicity. Mol Ther (2012) 0.98
Efficient gene targeting mediated by a lentiviral vector-associated meganuclease. Nucleic Acids Res (2011) 0.98
Concise review: lessons learned from clinical trials of gene therapy in monogenic immunodeficiency diseases. Stem Cells Transl Med (2014) 0.97
Creating and evaluating accurate CRISPR-Cas9 scalpels for genomic surgery. Nat Methods (2015) 0.96
Reactive oxygen species regulate hematopoietic stem cell self-renewal, migration and development, as well as their bone marrow microenvironment. Antioxid Redox Signal (2014) 0.96
High-throughput, sensitive quantification of repopulating hematopoietic stem cell clones. J Virol (2010) 0.95
Evaluating a ligation-mediated PCR and pyrosequencing method for the detection of clonal contribution in polyclonal retrovirally transduced samples. Hum Gene Ther Methods (2013) 0.95
Lentiviral transduction of CD34(+) cells induces genome-wide epigenetic modifications. PLoS One (2012) 0.95
Regulated apoptosis of genetically modified hematopoietic stem and progenitor cells via an inducible caspase-9 suicide gene in rhesus macaques. Stem Cells (2015) 0.94
Gene therapy: progress and predictions. Proc Biol Sci (2015) 0.94
VISPA: a computational pipeline for the identification and analysis of genomic vector integration sites. Genome Med (2014) 0.94
Gene therapy for hemoglobinopathies: the state of the field and the future. Hematol Oncol Clin North Am (2014) 0.94
Toxicity and management in CAR T-cell therapy. Mol Ther Oncolytics (2016) 0.93
Overexpression of EVI1 interferes with cytokinesis and leads to accumulation of cells with supernumerary centrosomes in G0/1 phase. Cell Cycle (2012) 0.92
Retargeting transposon insertions by the adeno-associated virus Rep protein. Nucleic Acids Res (2012) 0.92
Retroviral vectors: post entry events and genomic alterations. Viruses (2011) 0.92
Estimated comparative integration hotspots identify different behaviors of retroviral gene transfer vectors. PLoS Comput Biol (2011) 0.92
Adoptive T-cell therapy: adverse events and safety switches. Clin Transl Immunology (2014) 0.91
Targeted gene therapies: tools, applications, optimization. Crit Rev Biochem Mol Biol (2012) 0.91
Gammaretroviral vectors: biology, technology and application. Viruses (2011) 0.91
Retroviral gene therapy for X-linked chronic granulomatous disease: results from phase I/II trial. Mol Ther (2011) 0.91
Preclinical demonstration of lentiviral vector-mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency. Mol Ther (2013) 0.90
Liver-Directed Adeno-Associated Viral Gene Therapy for Hemophilia. J Genet Syndr Gene Ther (2012) 0.90
Development of gene therapy for blood disorders: an update. Blood (2013) 0.90
CRISPR-mediated genotypic and phenotypic correction of a chronic granulomatous disease mutation in human iPS cells. Exp Hematol (2015) 0.90
Impact of next-generation sequencing error on analysis of barcoded plasmid libraries of known complexity and sequence. Nucleic Acids Res (2014) 0.90
Ex vivo expansion of retrovirally transduced primate CD34+ cells results in overrepresentation of clones with MDS1/EVI1 insertion sites in the myeloid lineage after transplantation. Mol Ther (2010) 0.89
Charting a clear path: the ASGCT Standardized Pathways Conference. Mol Ther (2014) 0.89
Transduction of human CD34+ repopulating cells with a self-inactivating lentiviral vector for SCID-X1 produced at clinical scale by a stable cell line. Hum Gene Ther Methods (2012) 0.89
Clinical applications of gene therapy for primary immunodeficiencies. Hum Gene Ther (2015) 0.88
Generation of functional neutrophils from a mouse model of X-linked chronic granulomatous disorder using induced pluripotent stem cells. PLoS One (2011) 0.88
Alpharetroviral vector-mediated gene therapy for X-CGD: functional correction and lack of aberrant splicing. Mol Ther (2012) 0.88
Retroviral insertional mutagenesis can contribute to immortalization of mature T lymphocytes. Mol Med (2011) 0.88
Tracking gene and cell fate for therapeutic gain. Nat Mater (2014) 0.88
AAV-mediated liver-directed gene therapy. Methods Mol Biol (2011) 0.88
A Graph Based Framework to Model Virus Integration Sites. Comput Struct Biotechnol J (2015) 0.88
Efficient designer nuclease-based homologous recombination enables direct PCR screening for footprintless targeted human pluripotent stem cells. Stem Cell Reports (2014) 0.88
Genetically modified T cells for the treatment of malignant disease. Transfus Med Hemother (2013) 0.87
Correction of murine Rag2 severe combined immunodeficiency by lentiviral gene therapy using a codon-optimized RAG2 therapeutic transgene. Mol Ther (2012) 0.87
Complex or monosomal karyotype and not blast percentage is associated with poor survival in acute myeloid leukemia and myelodysplastic syndrome patients with inv(3)(q21q26.2)/t(3;3)(q21;q26.2): a Bone Marrow Pathology Group study. Haematologica (2014) 0.86
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LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science (2003) 19.77
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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
A mechanism linking extra centrosomes to chromosomal instability. Nature (2009) 9.45
Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J Clin Invest (2008) 9.42
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
Gene therapy for immunodeficiency due to adenosine deaminase deficiency. N Engl J Med (2009) 8.20
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
Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. Lancet (2004) 5.43
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Murine leukemia induced by retroviral gene marking. Science (2002) 3.59
Insertional mutagenesis identifies genes that promote the immortalization of primary bone marrow progenitor cells. Blood (2005) 2.93
Downregulation of histone H3 lysine 9 methyltransferase G9a induces centrosome disruption and chromosome instability in cancer cells. PLoS One (2008) 2.52
Recurrent retroviral vector integration at the Mds1/Evi1 locus in nonhuman primate hematopoietic cells. Blood (2005) 2.27
Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning. Mol Ther (2006) 2.08
Focusing on foci: H2AX and the recruitment of DNA-damage response factors. Cell Cycle (2003) 2.06
High EVI1 expression predicts poor survival in acute myeloid leukemia: a study of 319 de novo AML patients. Blood (2002) 2.01
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Impaired function of primitive hematopoietic cells in mice lacking the Mixed-Lineage-Leukemia homolog MLL5. Blood (2008) 1.80
Loss of MLL5 results in pleiotropic hematopoietic defects, reduced neutrophil immune function, and extreme sensitivity to DNA demethylation. Blood (2008) 1.79
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An unbiased genome-wide analysis of zinc-finger nuclease specificity. Nat Biotechnol (2011) 6.44
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Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. Science (2013) 5.64
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A differentiation checkpoint limits hematopoietic stem cell self-renewal in response to DNA damage. Cell (2012) 3.21
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Stem-cell gene therapy for the Wiskott-Aldrich syndrome. N Engl J Med (2010) 3.09
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Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity. Blood (2006) 3.07