Published in Blood on July 06, 2006
Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. J Clin Invest (2008) 7.05
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
Insertional transformation of hematopoietic cells by self-inactivating lentiviral and gammaretroviral vectors. Mol Ther (2009) 2.56
Genome-wide high-throughput integrome analyses by nrLAM-PCR and next-generation sequencing. Nat Protoc (2010) 2.24
Comprehensive genomic access to vector integration in clinical gene therapy. Nat Med (2009) 2.19
Ex vivo gene transfer and correction for cell-based therapies. Nat Rev Genet (2011) 2.13
Retroviral vector insertion sites associated with dominant hematopoietic clones mark "stemness" pathways. Blood (2006) 1.98
Gene therapy returns to centre stage. Nature (2015) 1.95
Efficient construction of producer cell lines for a SIN lentiviral vector for SCID-X1 gene therapy by concatemeric array transfection. Blood (2009) 1.73
Safe harbours for the integration of new DNA in the human genome. Nat Rev Cancer (2011) 1.67
Pharmacological targeting of the thrombomodulin-activated protein C pathway mitigates radiation toxicity. Nat Med (2012) 1.58
An experimental system for the evaluation of retroviral vector design to diminish the risk for proto-oncogene activation. Blood (2007) 1.57
Genotoxic potential of lineage-specific lentivirus vectors carrying the beta-globin locus control region. Mol Ther (2009) 1.46
Insertional gene activation by lentiviral and gammaretroviral vectors. J Virol (2008) 1.35
A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cells. Blood (2010) 1.33
Gene therapy for mucopolysaccharidosis. Expert Opin Biol Ther (2007) 1.28
β-globin gene transfer to human bone marrow for sickle cell disease. J Clin Invest (2013) 1.22
Mechanism of reduction in titers from lentivirus vectors carrying large inserts in the 3'LTR. Mol Ther (2009) 1.22
Cell-intrinsic and vector-related properties cooperate to determine the incidence and consequences of insertional mutagenesis. Mol Ther (2009) 1.21
Viral vectors: from virology to transgene expression. Br J Pharmacol (2009) 1.19
Sleeping beauty transposition from nonintegrating lentivirus. Mol Ther (2009) 1.18
In vivo biosafety model to assess the risk of adverse events from retroviral and lentiviral vectors. Mol Ther (2008) 1.14
Quantification of lentiviral vector copy numbers in individual hematopoietic colony-forming cells shows vector dose-dependent effects on the frequency and level of transduction. Gene Ther (2010) 1.14
Genomic and functional assays demonstrate reduced gammaretroviral vector genotoxicity associated with use of the cHS4 chromatin insulator. Mol Ther (2009) 1.13
Reduced genotoxicity of avian sarcoma leukosis virus vectors in rhesus long-term repopulating cells compared to standard murine retrovirus vectors. Mol Ther (2008) 1.12
The use of chromatin insulators to improve the expression and safety of integrating gene transfer vectors. Hum Gene Ther (2011) 1.11
Hematopoietic stem cell engineering at a crossroads. Blood (2011) 1.10
Gene therapy on the move. EMBO Mol Med (2013) 1.10
The 3' region of the chicken hypersensitive site-4 insulator has properties similar to its core and is required for full insulator activity. PLoS One (2009) 1.07
Highly efficient large-scale lentiviral vector concentration by tandem tangential flow filtration. J Virol Methods (2011) 1.06
Immunization with a lentivector that targets tumor antigen expression to dendritic cells induces potent CD8+ and CD4+ T-cell responses. J Virol (2007) 1.06
Stem cell gene therapy: the risks of insertional mutagenesis and approaches to minimize genotoxicity. Front Med (2011) 1.05
Interfering RNA-mediated purine analog resistance for in vitro and in vivo cell selection. Blood (2008) 1.04
Creating higher titer lentivirus with caffeine. Hum Gene Ther (2010) 1.01
Self-inactivating alpharetroviral vectors with a split-packaging design. J Virol (2010) 1.00
Alpharetroviral self-inactivating vectors: long-term transgene expression in murine hematopoietic cells and low genotoxicity. Mol Ther (2012) 0.98
Stem cell marking with promotor-deprived self-inactivating retroviral vectors does not lead to induced clonal imbalance. Mol Ther (2008) 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
Rescue of pyruvate kinase deficiency in mice by gene therapy using the human isoenzyme. Mol Ther (2009) 0.97
Concise review: lessons learned from clinical trials of gene therapy in monogenic immunodeficiency diseases. Stem Cells Transl Med (2014) 0.97
Finding the needle in the hay stack: hematopoietic stem cells in Fanconi anemia. Mutat Res (2009) 0.96
Preventing and exploiting the oncogenic potential of integrating gene vectors. J Clin Invest (2009) 0.95
Effect of the internal promoter on insertional gene activation by lentiviral vectors with an intact HIV long terminal repeat. 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
Contributions of gene marking to cell and gene therapies. Hum Gene Ther (2011) 0.95
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
Lentiviral vectors for cancer immunotherapy and clinical applications. Cancers (Basel) (2013) 0.93
Reciprocal relationship between O6-methylguanine-DNA methyltransferase P140K expression level and chemoprotection of hematopoietic stem cells. Cancer Res (2008) 0.92
Retroviral vectors: post entry events and genomic alterations. Viruses (2011) 0.92
Live and let die: in vivo selection of gene-modified hematopoietic stem cells via MGMT-mediated chemoprotection. DNA Repair (Amst) (2007) 0.92
Scale-up and manufacturing of clinical-grade self-inactivating γ-retroviral vectors by transient transfection. Gene Ther (2011) 0.92
Gammaretroviral vectors: biology, technology and application. Viruses (2011) 0.91
Self-inactivating retroviral vector-mediated gene transfer induces oncogene activation and immortalization of primary murine bone marrow cells. Mol Ther (2009) 0.91
Development of lentiviral gene therapy for Wiskott Aldrich syndrome. Expert Opin Biol Ther (2008) 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
Assessing the risks of genotoxicity in the therapeutic development of induced pluripotent stem cells. Mol Ther (2012) 0.90
Vector design for expression of O6-methylguanine-DNA methyltransferase in hematopoietic cells. DNA Repair (Amst) (2007) 0.90
Retroviral integration process in the human genome: is it really non-random? A new statistical approach. PLoS Comput Biol (2008) 0.89
Charting a clear path: the ASGCT Standardized Pathways Conference. Mol Ther (2014) 0.89
Retroviral vector integration in post-transplant hematopoiesis in mice conditioned with either submyeloablative or ablative irradiation. Gene Ther (2009) 0.89
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
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
Correction of murine SCID-X1 by lentiviral gene therapy using a codon-optimized IL2RG gene and minimal pretransplant conditioning. Mol Ther (2011) 0.88
Evaluation of residual promoter activity in γ-retroviral self-inactivating (SIN) vectors. Mol Ther (2011) 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
Improving TCR Gene Therapy for Treatment of Haematological Malignancies. Adv Hematol (2012) 0.87
Hematopoietic stem cell gene therapy:assessing the relevance of preclinical models. Semin Hematol (2013) 0.86
Neonatal intravenous injection of a gammaretroviral vector has a low incidence of tumor induction in mice. Hum Gene Ther (2008) 0.86
Dual-regulated lentiviral vector for gene therapy of X-linked chronic granulomatosis. Mol Ther (2014) 0.85
Mouse transplant models for evaluating the oncogenic risk of a self-inactivating XSCID lentiviral vector. PLoS One (2013) 0.85
Lentiviral vectors for induction of self-differentiation and conditional ablation of dendritic cells. Gene Ther (2011) 0.85
Evaluation of γ-retroviral vectors that mediate the inducible expression of IL-12 for clinical application. J Immunother (2012) 0.85
Deletion of the LTR enhancer/promoter has no impact on the integration profile of MLV vectors in human hematopoietic progenitors. PLoS One (2013) 0.84
Generation of a lentiviral vector producer cell clone for human Wiskott-Aldrich syndrome gene therapy. Mol Ther Methods Clin Dev (2015) 0.84
On target? Tracing zinc-finger-nuclease specificity. Nat Methods (2011) 0.84
Tracking of specific integrant clones in dogs treated with foamy virus vectors. Hum Gene Ther (2010) 0.84
Treatment of canine leukocyte adhesion deficiency by foamy virus vectors expressing CD18 from a PGK promoter. Gene Ther (2011) 0.83
The retroviral vector family: something for everyone. Virus Genes (2017) 0.83
Stably integrated and expressed retroviral sequences can influence nuclear location and chromatin condensation of the integration locus. Chromosoma (2012) 0.82
Cell- and gene-based therapeutic approaches for neurological deficits in mucopolysaccharidoses. Curr Pharm Biotechnol (2011) 0.82
Assessing the risk of T-cell malignancies in mouse models of SCID-X1. Mol Ther (2010) 0.82
The fetal mouse is a sensitive genotoxicity model that exposes lentiviral-associated mutagenesis resulting in liver oncogenesis. Mol Ther (2012) 0.82
Development of gene therapy: potential in severe combined immunodeficiency due to adenosine deaminase deficiency. Stem Cells Cloning (2009) 0.82
Viral hybrid vectors for somatic integration - are they the better solution? Viruses (2009) 0.81
Co-expression of MGMT(P140K) and alpha-L-iduronidase in primary hepatocytes from mucopolysaccharidosis type I mice enables efficient selection with metabolic correction. J Gene Med (2008) 0.80
Recent advances in gene therapy for severe congenital immunodeficiency diseases. Curr Opin Hematol (2008) 0.80
Retroviral and lentiviral vectors for the induction of immunological tolerance. Scientifica (Cairo) (2012) 0.80
Efficiency and safety of O⁶-methylguanine DNA methyltransferase (MGMT(P140K))-mediated in vivo selection in a humanized mouse model. Hum Gene Ther (2014) 0.80
Potentially therapeutic levels of anti-sickling globin gene expression following lentivirus-mediated gene transfer in sickle cell disease bone marrow CD34+ cells. Exp Hematol (2015) 0.79
Delivery of nucleic acid therapeutics by genetically engineered hematopoietic stem cells. Adv Drug Deliv Rev (2010) 0.79
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
Lack of genotoxicity due to foamy virus vector integration in human iPSCs. Gene Ther (2013) 0.78
Knockdown of HPRT for selection of genetically modified human hematopoietic progenitor cells. PLoS One (2013) 0.78
Update on clinical gene therapy in childhood. Arch Dis Child (2007) 0.78
Use of the piggyBac transposon to create stable packaging cell lines for the production of clinical-grade self-inactivating γ-retroviral vectors. Hum Gene Ther Methods (2014) 0.78
Strategies to insulate lentiviral vector-expressed transgenes. Methods Mol Biol (2010) 0.77
LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science (2003) 19.77
Plat-E: an efficient and stable system for transient packaging of retroviruses. Gene Ther (2000) 12.54
Transcription start regions in the human genome are favored targets for MLV integration. Science (2003) 12.21
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
Progress and problems with the use of viral vectors for gene therapy. Nat Rev Genet (2003) 6.95
Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science (2002) 6.46
Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. N Engl J Med (2002) 6.25
Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. Lancet (2004) 5.43
RTCGD: retroviral tagged cancer gene database. Nucleic Acids Res (2004) 4.41
Murine leukemia induced by retroviral gene marking. Science (2002) 3.59
Clonal dominance of hematopoietic stem cells triggered by retroviral gene marking. Science (2005) 3.58
Distinct genomic integration of MLV and SIV vectors in primate hematopoietic stem and progenitor cells. PLoS Biol (2004) 3.48
Insertional mutagenesis identifies genes that promote the immortalization of primary bone marrow progenitor cells. Blood (2005) 2.93
Polyclonal long-term repopulating stem cell clones in a primate model. Blood (2002) 2.53
Side effects of retroviral gene transfer into hematopoietic stem cells. Blood (2003) 2.35
Recurrent retroviral vector integration at the Mds1/Evi1 locus in nonhuman primate hematopoietic cells. Blood (2005) 2.27
Retroviral insertional mutagenesis: tagging cancer pathways. Adv Cancer Res (2003) 2.07
A chromatin insulator protects retrovirus vectors from chromosomal position effects. Proc Natl Acad Sci U S A (2000) 1.97
Detection and direct genomic sequencing of multiple rare unknown flanking DNA in highly complex samples. Hum Gene Ther (2001) 1.83
Leukemias following retroviral transfer of multidrug resistance 1 (MDR1) are driven by combinatorial insertional mutagenesis. Blood (2005) 1.82
Equal potency of gammaretroviral and lentiviral SIN vectors for expression of O6-methylguanine-DNA methyltransferase in hematopoietic cells. Mol Ther (2005) 1.82
Acute myeloid leukemia is associated with retroviral gene transfer to hematopoietic progenitor cells in a rhesus macaque. Blood (2006) 1.78
Context dependence of different modules for posttranscriptional enhancement of gene expression from retroviral vectors. Mol Ther (2000) 1.71
EVI1 and hematopoietic disorders: history and perspectives. Gene (2005) 1.44
Inhibition of human immunodeficiency virus type 1 entry in cells expressing gp41-derived peptides. J Virol (2004) 1.43
Sox4 cooperates with Evi1 in AKXD-23 myeloid tumors via transactivation of proviral LTR. Blood (2005) 1.34
Predictable and efficient retroviral gene transfer into murine bone marrow repopulating cells using a defined vector dose. Exp Hematol (2003) 1.16
HIV-1-derived lentiviral vectors. Curr Top Microbiol Immunol (2002) 1.10
Distinct classes of factor-independent mutants can be isolated after retroviral mutagenesis of a human myeloid stem cell line. Growth Factors (1993) 1.08
Gene therapy progress and prospects: novel gene therapy approaches for AIDS. Gene Ther (2005) 0.96
Protection and selection for gene therapy in the hematopoietic system. J Gene Med (2004) 0.91
International network of cancer genome projects. Nature (2010) 20.35
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
An unbiased genome-wide analysis of zinc-finger nuclease specificity. Nat Biotechnol (2011) 6.44
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
Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science (2013) 5.34
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
High-resolution insertion-site analysis by linear amplification-mediated PCR (LAM-PCR). Nat Methods (2007) 3.90
Murine leukemia induced by retroviral gene marking. Science (2002) 3.59
Clonal dominance of hematopoietic stem cells triggered by retroviral gene marking. Science (2005) 3.58
Distinct genomic integration of MLV and SIV vectors in primate hematopoietic stem and progenitor cells. PLoS Biol (2004) 3.48
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
Loss of interleukin-10 signaling and infantile inflammatory bowel disease: implications for diagnosis and therapy. Gastroenterology (2012) 3.28
Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy. J Clin Invest (2007) 3.28
A differentiation checkpoint limits hematopoietic stem cell self-renewal in response to DNA damage. Cell (2012) 3.21
Stem-cell gene therapy for the Wiskott-Aldrich syndrome. N Engl J Med (2010) 3.09
Integrative genomic analyses reveal an androgen-driven somatic alteration landscape in early-onset prostate cancer. Cancer Cell (2013) 3.08
Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo. J Clin Invest (2007) 2.80
Hot spots of retroviral integration in human CD34+ hematopoietic cells. Blood (2007) 2.79
Effective gene therapy with nonintegrating lentiviral vectors. Nat Med (2006) 2.70
Physiological promoters reduce the genotoxic risk of integrating gene vectors. Mol Ther (2008) 2.56
Insertional transformation of hematopoietic cells by self-inactivating lentiviral and gammaretroviral vectors. Mol Ther (2009) 2.56
Polyclonal long-term repopulating stem cell clones in a primate model. Blood (2002) 2.53
Distinct types of tumor-initiating cells form human colon cancer tumors and metastases. Cell Stem Cell (2011) 2.47
A largely random AAV integration profile after LPLD gene therapy. Nat Med (2013) 2.42
Recurrent somatic alterations of FGFR1 and NTRK2 in pilocytic astrocytoma. Nat Genet (2013) 2.42
Side effects of retroviral gene transfer into hematopoietic stem cells. Blood (2003) 2.35
Resistance of mature T cells to oncogene transformation. Blood (2008) 2.32
Recurrent retroviral vector integration at the Mds1/Evi1 locus in nonhuman primate hematopoietic cells. Blood (2005) 2.27
BRAF inhibition in refractory hairy-cell leukemia. N Engl J Med (2012) 2.25
Genome-wide high-throughput integrome analyses by nrLAM-PCR and next-generation sequencing. Nat Protoc (2010) 2.24
Phase 2 gene therapy trial of an anti-HIV ribozyme in autologous CD34+ cells. Nat Med (2009) 2.21
A hybrid vector for ligand-directed tumor targeting and molecular imaging. Cell (2006) 2.20
Comprehensive genomic access to vector integration in clinical gene therapy. Nat Med (2009) 2.19
Overcoming reprogramming resistance of Fanconi anemia cells. Blood (2012) 2.07
Temperature triggered self-assembly of polypeptides into multivalent spherical micelles. J Am Chem Soc (2007) 2.01
Bone marrow-derived cells contribute to infarct remodelling. Cardiovasc Res (2006) 2.00
Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity. Sci Transl Med (2014) 2.00
Retroviral vector insertion sites associated with dominant hematopoietic clones mark "stemness" pathways. Blood (2006) 1.98
Lentiviral vectors pseudotyped with murine ecotropic envelope: increased biosafety and convenience in preclinical research. Exp Hematol (2006) 1.95
TALEN-based gene correction for epidermolysis bullosa. Mol Ther (2013) 1.84
The phenotype of human STK4 deficiency. Blood (2012) 1.84
Leukemias following retroviral transfer of multidrug resistance 1 (MDR1) are driven by combinatorial insertional mutagenesis. Blood (2005) 1.82
Equal potency of gammaretroviral and lentiviral SIN vectors for expression of O6-methylguanine-DNA methyltransferase in hematopoietic cells. Mol Ther (2005) 1.82
Stem cell collection and gene transfer in Fanconi anemia. Mol Ther (2007) 1.81
Acute myeloid leukemia is associated with retroviral gene transfer to hematopoietic progenitor cells in a rhesus macaque. Blood (2006) 1.78
Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection. Blood (2011) 1.76
Lentiviral vector design and imaging approaches to visualize the early stages of cellular reprogramming. Mol Ther (2011) 1.69
Hepatocyte-targeted expression by integrase-defective lentiviral vectors induces antigen-specific tolerance in mice with low genotoxic risk. Hepatology (2011) 1.66
NAMPT is essential for the G-CSF-induced myeloid differentiation via a NAD(+)-sirtuin-1-dependent pathway. Nat Med (2009) 1.66
Clonality analysis after retroviral-mediated gene transfer to CD34+ cells from the cord blood of ADA-deficient SCID neonates. Nat Med (2003) 1.65
Chance or necessity? Insertional mutagenesis in gene therapy and its consequences. Mol Ther (2004) 1.65
HOXB4 enforces equivalent fates of ES-cell-derived and adult hematopoietic cells. Proc Natl Acad Sci U S A (2005) 1.65
Safety and liver transduction efficacy of rAAV5-cohPBGD in nonhuman primates: a potential therapy for acute intermittent porphyria. Hum Gene Ther (2013) 1.62