Published in Mol Ther on October 01, 2005
A transposon and transposase system for human application. Mol Ther (2010) 2.28
Lentiviral vectors with a defective integrase allow efficient and sustained transgene expression in vitro and in vivo. Proc Natl Acad Sci U S A (2006) 1.74
Lentiviral delivery of short hairpin RNAs. Adv Drug Deliv Rev (2009) 1.65
Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy. J Clin Invest (2015) 1.46
Lentiviral vectors in gene therapy: their current status and future potential. Arch Immunol Ther Exp (Warsz) (2010) 1.29
Gene therapy for mucopolysaccharidosis. Expert Opin Biol Ther (2007) 1.28
A look to future directions in gene therapy research for monogenic diseases. PLoS Genet (2006) 1.11
Functional expression of Rab escort protein 1 following AAV2-mediated gene delivery in the retina of choroideremia mice and human cells ex vivo. J Mol Med (Berl) (2013) 1.09
Lentiviral vector-based insertional mutagenesis identifies genes associated with liver cancer. Nat Methods (2013) 1.08
Predicting preferential DNA vector insertion sites: implications for functional genomics and gene therapy. Genome Biol (2007) 1.08
Correction of ADAMTS13 deficiency by in utero gene transfer of lentiviral vector encoding ADAMTS13 genes. Mol Ther (2008) 1.05
Corticospinal tract transduction: a comparison of seven adeno-associated viral vector serotypes and a non-integrating lentiviral vector. Gene Ther (2011) 1.03
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
A helper-dependent capsid-modified adenovirus vector expressing adeno-associated virus rep78 mediates site-specific integration of a 27-kilobase transgene cassette. J Virol (2006) 0.97
Retroviral vectors: post entry events and genomic alterations. Viruses (2011) 0.92
Erythroid-specific expression of beta-globin by the sleeping beauty transposon for Sickle cell disease. Biochemistry (2007) 0.92
LDLR-Gene therapy for familial hypercholesterolaemia: problems, progress, and perspectives. Int Arch Med (2010) 0.91
Vector design for expression of O6-methylguanine-DNA methyltransferase in hematopoietic cells. DNA Repair (Amst) (2007) 0.90
Clinical applications involving CNS gene transfer. Adv Genet (2014) 0.89
Methods for gene transfer to the central nervous system. Adv Genet (2014) 0.88
Epstein-Barr virus nuclear antigen 1 does not cause lymphoma in C57BL/6J mice. J Virol (2008) 0.87
Neonatal intravenous injection of a gammaretroviral vector has a low incidence of tumor induction in mice. Hum Gene Ther (2008) 0.86
Neonatal gene therapy of glycogen storage disease type Ia using a feline immunodeficiency virus-based vector. Mol Ther (2010) 0.84
Reducing the genotoxic potential of retroviral vectors. Methods Mol Biol (2008) 0.84
Perinatal gene transfer to the liver. Curr Pharm Des (2011) 0.84
Successful gene therapy in utero for lethal murine hypophosphatasia. Hum Gene Ther (2012) 0.84
Impact of hydrodynamic injection and phiC31 integrase on tumor latency in a mouse model of MYC-induced hepatocellular carcinoma. PLoS One (2010) 0.84
Mesenchymal stem cells as a gene delivery vehicle for successful islet transplantation. Pharm Res (2011) 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
The fetal mouse is a sensitive genotoxicity model that exposes lentiviral-associated mutagenesis resulting in liver oncogenesis. Mol Ther (2012) 0.82
Transduction of fetal mice with a feline lentiviral vector induces liver tumors which exhibit an E2F activation signature. Mol Ther (2013) 0.79
Lentiviral vector-mediated RNA silencing in the central nervous system. Hum Gene Ther Methods (2013) 0.79
Virally mediated gene manipulation in the adult CNS. Front Mol Neurosci (2011) 0.79
The fetal respiratory system as target for antenatal therapy. Facts Views Vis Obgyn (2011) 0.79
Engraftment potential of adipose tissue-derived human mesenchymal stem cells after transplantation in the fetal rabbit. Stem Cells Dev (2012) 0.78
Use of integrase-minus lentiviral vector for transient expression. Cell J (2012) 0.77
Perinatal systemic gene delivery using adeno-associated viral vectors. Front Mol Neurosci (2014) 0.77
In utero stem cell transplantation and gene therapy: rationale, history, and recent advances toward clinical application. Mol Ther Methods Clin Dev (2016) 0.76
A method to estimate the efficiency of gene expression from an integrated retroviral vector. Retrovirology (2006) 0.76
Effects of vector backbone and pseudotype on lentiviral vector-mediated gene transfer: studies in infant ADA-deficient mice and rhesus monkeys. Mol Ther (2014) 0.75
Induction of Immune Tolerance to Foreign Protein via Adeno-Associated Viral Vector Gene Transfer in Mid-Gestation Fetal Sheep. PLoS One (2017) 0.75
Lentivirus-meditated frataxin gene delivery reverses genome instability in Friedreich ataxia patient and mouse model fibroblasts. Gene Ther (2016) 0.75
International network of cancer genome projects. Nature (2010) 20.35
Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med (2008) 15.30
Broad antiretroviral defence by human APOBEC3G through lethal editing of nascent reverse transcripts. Nature (2003) 13.24
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
Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. N Engl J Med (2002) 6.25
Conditional suppression of cellular genes: lentivirus vector-mediated drug-inducible RNA interference. J Virol (2003) 6.21
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
Inhibition of hepatitis B virus replication by APOBEC3G. Science (2004) 5.15
Adenovirus serotype 5 hexon mediates liver gene transfer. Cell (2008) 5.00
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
KAP1 controls endogenous retroviruses in embryonic stem cells. Nature (2010) 4.02
Embryonic stem cell potency fluctuates with endogenous retrovirus activity. Nature (2012) 3.96
Genetic reactivation of cone photoreceptors restores visual responses in retinitis pigmentosa. Science (2010) 3.92
High-resolution insertion-site analysis by linear amplification-mediated PCR (LAM-PCR). Nat Methods (2007) 3.90
In embryonic stem cells, ZFP57/KAP1 recognize a methylated hexanucleotide to affect chromatin and DNA methylation of imprinting control regions. Mol Cell (2011) 3.67
Murine leukemia induced by retroviral gene marking. Science (2002) 3.59
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
Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy. J Clin Invest (2007) 3.28
Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins. Mol Ther (2011) 3.10
A robust model for read count data in exome sequencing experiments and implications for copy number variant calling. Bioinformatics (2012) 3.10
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
A versatile tool for conditional gene expression and knockdown. Nat Methods (2006) 2.92
A switch between topological domains underlies HoxD genes collinearity in mouse limbs. Science (2013) 2.86
Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo. J Clin Invest (2007) 2.80
Essential role of the NADPH oxidase subunit p47(phox) in endothelial cell superoxide production in response to phorbol ester and tumor necrosis factor-alpha. Circ Res (2002) 2.79
Hot spots of retroviral integration in human CD34+ hematopoietic cells. Blood (2007) 2.79
A single amino acid determinant governs the species-specific sensitivity of APOBEC3G to Vif action. J Biol Chem (2004) 2.79
Effective gene therapy with nonintegrating lentiviral vectors. Nat Med (2006) 2.70
Multiple vitamin K-dependent coagulation zymogens promote adenovirus-mediated gene delivery to hepatocytes. Blood (2006) 2.67
Insertional transformation of hematopoietic cells by self-inactivating lentiviral and gammaretroviral vectors. Mol Ther (2009) 2.56
Identification of coagulation factor (F)X binding sites on the adenovirus serotype 5 hexon: effect of mutagenesis on FX interactions and gene transfer. Blood (2009) 2.55
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
KRAB-zinc finger proteins and KAP1 can mediate long-range transcriptional repression through heterochromatin spreading. PLoS Genet (2010) 2.44
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
Characterization and clinical application of human CD34+ stem/progenitor cell populations mobilized into the blood by granulocyte colony-stimulating factor. Stem Cells (2006) 2.30
The Wiskott-Aldrich syndrome. J Allergy Clin Immunol (2006) 2.29
Recurrent retroviral vector integration at the Mds1/Evi1 locus in nonhuman primate hematopoietic cells. Blood (2005) 2.27
Clinical features and outcome of patients with IRAK-4 and MyD88 deficiency. Medicine (Baltimore) (2010) 2.26
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
KAP1-mediated epigenetic repression in the forebrain modulates behavioral vulnerability to stress. Neuron (2008) 2.17
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
Temperature triggered self-assembly of polypeptides into multivalent spherical micelles. J Am Chem Soc (2007) 2.01
Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity. Sci Transl Med (2014) 2.00