Published in Science on November 06, 2009
Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia. Nature (2010) 6.95
Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo. Nat Biotechnol (2010) 6.55
Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. Science (2013) 5.64
In vivo genome editing restores haemostasis in a mouse model of haemophilia. Nature (2011) 4.81
Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease. Nat Med (2010) 4.76
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Retroviral integrations in gene therapy trials. Mol Ther (2012) 1.34
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20 years of gene therapy for SCID. Nat Immunol (2010) 1.33
Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model. Blood (2010) 1.33
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β-globin gene transfer to human bone marrow for sickle cell disease. J Clin Invest (2013) 1.22
Progress in gene therapy for neurological disorders. Nat Rev Neurol (2013) 1.22
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Replacement of brain-resident myeloid cells does not alter cerebral amyloid-β deposition in mouse models of Alzheimer's disease. J Exp Med (2015) 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
Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors. Nat Biotechnol (2015) 1.17
Human oxidation-specific antibodies reduce foam cell formation and atherosclerosis progression. J Am Coll Cardiol (2011) 1.16
Clonal tracking of rhesus macaque hematopoiesis highlights a distinct lineage origin for natural killer cells. Cell Stem Cell (2014) 1.15
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
Uncovering and dissecting the genotoxicity of self-inactivating lentiviral vectors in vivo. Mol Ther (2014) 1.13
A TLR and non-TLR mediated innate response to lentiviruses restricts hepatocyte entry and can be ameliorated by pharmacological blockade. Mol Ther (2012) 1.13
Two decades of clinical gene therapy--success is finally mounting. Discov Med (2010) 1.13
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Brain conditioning is instrumental for successful microglia reconstitution following hematopoietic stem cell transplantation. Proc Natl Acad Sci U S A (2012) 1.12
Nup153 and Nup98 bind the HIV-1 core and contribute to the early steps of HIV-1 replication. Virology (2013) 1.11
A microRNA-regulated and GP64-pseudotyped lentiviral vector mediates stable expression of FVIII in a murine model of Hemophilia A. Mol Ther (2011) 1.11
Hematopoietic stem cell engineering at a crossroads. Blood (2011) 1.10
Correction of beta-thalassemia major by gene transfer in haematopoietic progenitors of pediatric patients. EMBO Mol Med (2010) 1.10
Analyzing the number of common integration sites of viral vectors--new methods and computer programs. PLoS One (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
Targeted gene addition in human epithelial stem cells by zinc-finger nuclease-mediated homologous recombination. Mol Ther (2013) 1.10
Advances in Gene Delivery Systems. Pharmaceut Med (2011) 1.09
Pathophysiology of X-linked adrenoleukodystrophy. Biochimie (2013) 1.09
Integrase-defective lentiviral vectors as a delivery platform for targeted modification of adenosine deaminase locus. Mol Ther (2013) 1.08
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
Macrophage-mediated GDNF delivery protects against dopaminergic neurodegeneration: a therapeutic strategy for Parkinson's disease. Mol Ther (2010) 1.07
Induced pluripotent mesenchymal stromal cell clones retain donor-derived differences in DNA methylation profiles. Mol Ther (2012) 1.07
Engineering biomaterial systems to enhance viral vector gene delivery. Mol Ther (2011) 1.07
A new generation of human artificial chromosomes for functional genomics and gene therapy. Cell Mol Life Sci (2012) 1.06
Highly efficient large-scale lentiviral vector concentration by tandem tangential flow filtration. J Virol Methods (2011) 1.06
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
Stem cell gene therapy: the risks of insertional mutagenesis and approaches to minimize genotoxicity. Front Med (2011) 1.05
Gene correction by homologous recombination with zinc finger nucleases in primary cells from a mouse model of a generic recessive genetic disease. Mol Ther (2010) 1.04
Development of B-lineage predominant lentiviral vectors for use in genetic therapies for B cell disorders. Mol Ther (2010) 1.04
Rapamycin relieves lentiviral vector transduction resistance in human and mouse hematopoietic stem cells. Blood (2014) 1.03
Generation of healthy mice from gene-corrected disease-specific induced pluripotent stem cells. PLoS Biol (2011) 1.02
Chromatin landscapes of retroviral and transposon integration profiles. PLoS Genet (2014) 1.02
Dynamics of HSPC repopulation in nonhuman primates revealed by a decade-long clonal-tracking study. Cell Stem Cell (2014) 1.02
Zinc-finger-based transcriptional repression of rhodopsin in a model of dominant retinitis pigmentosa. EMBO Mol Med (2011) 1.02
Gene therapy matures in the clinic. Nat Biotechnol (2012) 1.02
Microglia across the lifespan: from origin to function in brain development, plasticity and cognition. J Physiol (2016) 1.02
Lentiviral vector induced insertional haploinsufficiency of Ebf1 causes murine leukemia. Mol Ther (2012) 1.01
Creating higher titer lentivirus with caffeine. Hum Gene Ther (2010) 1.01
Biosafety features of lentiviral vectors. Hum Gene Ther (2013) 1.01
Hematopoietic stem cell and gene therapy corrects primary neuropathology and behavior in mucopolysaccharidosis IIIA mice. Mol Ther (2012) 1.00
Modification of hematopoietic stem/progenitor cells with CD19-specific chimeric antigen receptors as a novel approach for cancer immunotherapy. Hum Gene Ther (2013) 1.00
Stable human FIX expression after 0.9G intrauterine gene transfer of self-complementary adeno-associated viral vector 5 and 8 in macaques. Mol Ther (2011) 1.00
Lentiviral vector gene therapy: effective and safe? Mol Ther (2010) 0.99
Variation in FTO contributes to childhood obesity and severe adult obesity. Nat Genet (2007) 13.62
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
A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N Engl J Med (2003) 8.59
Regression of splenic lymphoma with villous lymphocytes after treatment of hepatitis C virus infection. N Engl J Med (2002) 7.52
Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. J Clin Invest (2008) 7.05
Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia. Nature (2010) 6.95
An unbiased genome-wide analysis of zinc-finger nuclease specificity. Nat Biotechnol (2011) 6.44
Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. N Engl J Med (2002) 6.25
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
Cernunnos, a novel nonhomologous end-joining factor, is mutated in human immunodeficiency with microcephaly. Cell (2006) 5.33
Munc13-4 is essential for cytolytic granules fusion and is mutated in a form of familial hemophagocytic lymphohistiocytosis (FHL3). Cell (2003) 4.94
Gene therapy: therapeutic gene causing lymphoma. Nature (2006) 4.89
Chronic infantile neurological cutaneous and articular syndrome is caused by mutations in CIAS1, a gene highly expressed in polymorphonuclear cells and chondrocytes. Am J Hum Genet (2002) 4.84
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
Autoantibodies against IL-17A, IL-17F, and IL-22 in patients with chronic mucocutaneous candidiasis and autoimmune polyendocrine syndrome type I. J Exp Med (2010) 3.98
Mutations in STAT3 and IL12RB1 impair the development of human IL-17-producing T cells. J Exp Med (2008) 3.85
Human uracil-DNA glycosylase deficiency associated with profoundly impaired immunoglobulin class-switch recombination. Nat Immunol (2003) 3.81
Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trial. Lancet (2008) 3.74
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AID mutant analyses indicate requirement for class-switch-specific cofactors. Nat Immunol (2003) 3.69
B cell-helper neutrophils stimulate the diversification and production of immunoglobulin in the marginal zone of the spleen. Nat Immunol (2011) 3.66
Gain-of-function human STAT1 mutations impair IL-17 immunity and underlie chronic mucocutaneous candidiasis. J Exp Med (2011) 3.63
Insulin resistance in chronic hepatitis C: association with genotypes 1 and 4, serum HCV RNA level, and liver fibrosis. Gastroenterology (2007) 3.61
Murine leukemia induced by retroviral gene marking. Science (2002) 3.59
Efficacy of gene therapy for X-linked severe combined immunodeficiency. N Engl J Med (2010) 3.54
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Human immunodeficiency virus-driven expansion of CD4+CD25+ regulatory T cells, which suppress HIV-specific CD4 T-cell responses in HIV-infected patients. Blood (2004) 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
Phosphoinositide 3-kinase δ gene mutation predisposes to respiratory infection and airway damage. Science (2013) 3.33
XIAP deficiency in humans causes an X-linked lymphoproliferative syndrome. Nature (2006) 3.30
Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy. J Clin Invest (2007) 3.28
Inflammasome activation in NADPH oxidase defective mononuclear phagocytes from patients with chronic granulomatous disease. Blood (2010) 3.23
Allogeneic bone marrow transplantation in mevalonic aciduria. N Engl J Med (2007) 3.18
Angiogenic synergism, vascular stability and improvement of hind-limb ischemia by a combination of PDGF-BB and FGF-2. Nat Med (2003) 3.16
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
Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity. Blood (2006) 3.07
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Early serum HBsAg drop: a strong predictor of sustained virological response to pegylated interferon alfa-2a in HBeAg-negative patients. Hepatology (2009) 2.97
Mutations in GLIS3 are responsible for a rare syndrome with neonatal diabetes mellitus and congenital hypothyroidism. Nat Genet (2006) 2.95
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
Senataxin, the ortholog of a yeast RNA helicase, is mutant in ataxia-ocular apraxia 2. Nat Genet (2004) 2.75
ANRIL, a long, noncoding RNA, is an unexpected major hotspot in GWAS. FASEB J (2010) 2.73
STIM1 mutation associated with a syndrome of immunodeficiency and autoimmunity. N Engl J Med (2009) 2.72
Effective gene therapy with nonintegrating lentiviral vectors. Nat Med (2006) 2.70
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DNA bar coding and pyrosequencing to analyze adverse events in therapeutic gene transfer. Nucleic Acids Res (2008) 2.64
New ISSCR guidelines underscore major principles for responsible translational stem cell research. Cell Stem Cell (2008) 2.61
Non-synonymous polymorphisms in melanocortin-4 receptor protect against obesity: the two facets of a Janus obesity gene. Hum Mol Genet (2007) 2.50
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Early versus deferred antiretroviral multidrug therapy in infants infected with HIV type 1. Clin Infect Dis (2004) 2.46
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