Published in Hum Gene Ther on January 01, 2005
A nonviral minicircle vector for deriving human iPS cells. Nat Methods (2010) 4.42
IL-23 induces spondyloarthropathy by acting on ROR-γt+ CD3+CD4-CD8- entheseal resident T cells. Nat Med (2012) 4.38
The cytokines interleukin 27 and interferon-γ promote distinct Treg cell populations required to limit infection-induced pathology. Immunity (2012) 2.52
A robust system for production of minicircle DNA vectors. Nat Biotechnol (2010) 1.95
Novel minicircle vector for gene therapy in murine myocardial infarction. Circulation (2009) 1.45
Short hairpin RNA interference therapy for ischemic heart disease. Circulation (2008) 1.34
Generation of adult human induced pluripotent stem cells using nonviral minicircle DNA vectors. Nat Protoc (2010) 1.30
Derivation of human induced pluripotent stem cells for cardiovascular disease modeling. Circ Res (2011) 1.17
Double knockdown of prolyl hydroxylase and factor-inhibiting hypoxia-inducible factor with nonviral minicircle gene therapy enhances stem cell mobilization and angiogenesis after myocardial infarction. Circulation (2011) 1.14
Technological overview of iPS induction from human adult somatic cells. Curr Gene Ther (2013) 1.13
Gene doping: the hype and the reality. Br J Pharmacol (2008) 1.10
Minicircle DNA-based gene therapy coupled with immune modulation permits long-term expression of α-L-iduronidase in mice with mucopolysaccharidosis type I. Mol Ther (2010) 1.08
IL-23 is critical for induction of arthritis, osteoclast formation, and maintenance of bone mass. J Immunol (2011) 1.08
A mini-intronic plasmid (MIP): a novel robust transgene expression vector in vivo and in vitro. Mol Ther (2013) 1.05
Radioprotection in vitro and in vivo by minicircle plasmid carrying the human manganese superoxide dismutase transgene. Hum Gene Ther (2008) 1.04
New generation of plasmid backbones devoid of antibiotic resistance marker for gene therapy trials. Mol Ther (2011) 1.04
The Bacterial Ghost platform system: production and applications. Bioeng Bugs (2011) 1.00
Vectors for airway gene delivery. AAPS J (2007) 0.99
The extragenic spacer length between the 5' and 3' ends of the transgene expression cassette affects transgene silencing from plasmid-based vectors. Mol Ther (2012) 0.98
Nanoparticle applications in ocular gene therapy. Vision Res (2007) 0.98
pEPito: a significantly improved non-viral episomal expression vector for mammalian cells. BMC Biotechnol (2010) 0.97
Nanoparticles for retinal gene therapy. Prog Retin Eye Res (2010) 0.95
Minicircle DNA is superior to plasmid DNA in eliciting antigen-specific CD8+ T-cell responses. Mol Ther (2013) 0.93
Native homing endonucleases can target conserved genes in humans and in animal models. Nucleic Acids Res (2011) 0.90
Recombination-dependent oligomerization of human papillomavirus genomes upon transient DNA replication. J Virol (2013) 0.89
A novel micro-linear vector for in vitro and in vivo gene delivery and its application for EBV positive tumors. PLoS One (2012) 0.86
Advancement and prospects of tumor gene therapy. Chin J Cancer (2011) 0.85
Nonviral ocular gene therapy: assessment and future directions. Curr Opin Mol Ther (2008) 0.85
Efficient expression of vascular endothelial growth factor using minicircle DNA for angiogenic gene therapy. J Control Release (2007) 0.85
Minicircle-oriP-IFNγ: a novel targeted gene therapeutic system for EBV positive human nasopharyngeal carcinoma. PLoS One (2011) 0.84
Chemical chaperones improve protein secretion and rescue mutant factor VIII in mice with hemophilia A. PLoS One (2012) 0.82
Molecular-genetic imaging of cancer. Adv Cancer Res (2014) 0.81
A platform for rapid prototyping of synthetic gene networks in mammalian cells. Nucleic Acids Res (2014) 0.81
C17 prevents inflammatory arthritis and associated joint destruction in mice. PLoS One (2011) 0.81
Ultrasound directs a transposase system for durable hepatic gene delivery in mice. Ultrasound Med Biol (2013) 0.80
A new strategy to deliver synthetic protein drugs: self-reproducible biologics using minicircles. Sci Rep (2014) 0.80
Comparative study of nanoparticle-mediated transfection in different GI epithelium co-culture models. J Control Release (2012) 0.80
The myeloid receptor PILRβ mediates the balance of inflammatory responses through regulation of IL-27 production. PLoS One (2012) 0.79
Antibiotic-free selection in biotherapeutics: now and forever. Pathogens (2015) 0.79
Mechanism and methods to induce pluripotency. Protein Cell (2011) 0.78
A Dual Target-directed Agent against Interleukin-6 Receptor and Tumor Necrosis Factor α ameliorates experimental arthritis. Sci Rep (2016) 0.78
A novel in vivo gene transfer technique and in vitro cell based assays for the study of bone loss in musculoskeletal disorders. J Vis Exp (2014) 0.77
Applying horizontal gene transfer phenomena to enhance non-viral gene therapy. J Control Release (2013) 0.76
Nanoparticle-mediated rhodopsin cDNA but not intron-containing DNA delivery causes transgene silencing in a rhodopsin knockout model. FASEB J (2015) 0.76
Progress toward establishing embryonic stem or induced pluripotent stem cell-based clinical translation. Curr Opin Organ Transplant (2014) 0.76
A 5'non-coding exon containing engineered intron enhances transgene expression from recombinant AAV vectors in vivo. Hum Gene Ther (2016) 0.75
A Compendium of Preparation and Application of Stem Cells in Parkinson's Disease: Current Status and Future Prospects. Front Aging Neurosci (2016) 0.75
Sequence-Modified Antibiotic Resistance Genes Provide Sustained Plasmid-Mediated Transgene Expression in Mammals. Mol Ther (2017) 0.75
Minicircle Mediated Gene Delivery to Canine and Equine Mesenchymal Stem Cells. Int J Mol Sci (2017) 0.75
Ultrasound-targeted hepatic delivery of factor IX in hemophiliac mice. Gene Ther (2016) 0.75
Critical Role of LTB4/BLT1 in IL-23-Induced Synovial Inflammation and Osteoclastogenesis via NF-κB. J Immunol (2016) 0.75
Etanercept-Synthesising Mesenchymal Stem Cells Efficiently Ameliorate Collagen-Induced Arthritis. Sci Rep (2017) 0.75
MicroRNA-206 Prevents the Pathogenesis of Hepatocellular Carcinoma Via Modulating Expression of cMet and Cdk6. Hepatology (2017) 0.75
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med (2006) 13.51
Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med (2011) 11.01
Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways. Nature (2006) 10.95
Progress and problems with the use of viral vectors for gene therapy. Nat Rev Genet (2003) 6.95
RNA interference in adult mice. Nature (2002) 5.33
Robust expansion of human hepatocytes in Fah-/-/Rag2-/-/Il2rg-/- mice. Nat Biotechnol (2007) 5.04
Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8. Mol Ther (2006) 4.64
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood (2002) 4.60
A nonviral minicircle vector for deriving human iPS cells. Nat Methods (2010) 4.42
In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses. J Virol (2008) 3.79
Biological basis for restriction of microRNA targets to the 3' untranslated region in mammalian mRNAs. Nat Struct Mol Biol (2009) 3.70
Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors. J Virol (2004) 3.65
Human tRNA-derived small RNAs in the global regulation of RNA silencing. RNA (2010) 3.40
Sarcoma derived from cultured mesenchymal stem cells. Stem Cells (2006) 3.40
Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice. J Virol (2005) 3.28
Inhibition of hepatitis B virus in mice by RNA interference. Nat Biotechnol (2003) 3.15
High-resolution genome-wide mapping of transposon integration in mammals. Mol Cell Biol (2005) 3.10
Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo. Mol Ther (2003) 2.95
The 37/67-kilodalton laminin receptor is a receptor for adeno-associated virus serotypes 8, 2, 3, and 9. J Virol (2006) 2.94
Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med (2014) 2.92
Fate tracing of mature hepatocytes in mouse liver homeostasis and regeneration. J Clin Invest (2011) 2.83
AAV serotype 2 vectors preferentially integrate into active genes in mice. Nat Genet (2003) 2.81
Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy. Blood (2003) 2.74
Distinct pathways of genomic progression to benign and malignant tumors of the liver. Proc Natl Acad Sci U S A (2007) 2.56
Site-specific genomic integration produces therapeutic Factor IX levels in mice. Nat Biotechnol (2002) 2.54
Helper virus-free, optically controllable, and two-plasmid-based production of adeno-associated virus vectors of serotypes 1 to 6. Mol Ther (2003) 2.49
In vivo activity of nuclease-resistant siRNAs. RNA (2004) 2.33
Transposition from a gutless adeno-transposon vector stabilizes transgene expression in vivo. Nat Biotechnol (2002) 2.31
Large-scale molecular characterization of adeno-associated virus vector integration in mouse liver. J Virol (2005) 2.09
Expression of shRNA from a tissue-specific pol II promoter is an effective and safe RNAi therapeutic. Mol Ther (2008) 2.01
Mutational analysis of the N-terminal DNA-binding domain of sleeping beauty transposase: critical residues for DNA binding and hyperactivity in mammalian cells. Mol Cell Biol (2004) 1.97
A robust system for production of minicircle DNA vectors. Nat Biotechnol (2010) 1.95
Argonaute proteins are key determinants of RNAi efficacy, toxicity, and persistence in the adult mouse liver. J Clin Invest (2010) 1.91
The loop position of shRNAs and pre-miRNAs is critical for the accuracy of dicer processing in vivo. Cell (2012) 1.84
Genome editing of human embryonic stem cells and induced pluripotent stem cells with zinc finger nucleases for cellular imaging. Circ Res (2012) 1.84
Site-directed transposon integration in human cells. Nucleic Acids Res (2007) 1.74
A limited number of transducible hepatocytes restricts a wide-range linear vector dose response in recombinant adeno-associated virus-mediated liver transduction. J Virol (2002) 1.71
Adenovirus transduction is required for the correction of diabetes using Pdx-1 or Neurogenin-3 in the liver. Mol Ther (2007) 1.51
Determinants of hepatitis C translational initiation in vitro, in cultured cells and mice. Mol Ther (2002) 1.51
Histone modifications are associated with the persistence or silencing of vector-mediated transgene expression in vivo. Mol Ther (2007) 1.51
Silencing of episomal transgene expression in liver by plasmid bacterial backbone DNA is independent of CpG methylation. Mol Ther (2008) 1.49
Novel minicircle vector for gene therapy in murine myocardial infarction. Circulation (2009) 1.45
Helper-Independent Sleeping Beauty transposon-transposase vectors for efficient nonviral gene delivery and persistent gene expression in vivo. Mol Ther (2003) 1.42
Adeno-associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo. Hepatology (2010) 1.41
Combinatorial RNAi: a winning strategy for the race against evolving targets? Mol Ther (2007) 1.40
In vivo correction of murine tyrosinemia type I by DNA-mediated transposition. Mol Ther (2002) 1.39
Nonhomologous-end-joining factors regulate DNA repair fidelity during Sleeping Beauty element transposition in mammalian cells. Mol Cell Biol (2003) 1.37
Liver transduction with recombinant adeno-associated virus is primarily restricted by capsid serotype not vector genotype. J Virol (2006) 1.36
Immunogenicity and immunomodulatory properties of umbilical cord lining mesenchymal stem cells. Cell Transplant (2010) 1.36
The role of DNA-PKcs and artemis in opening viral DNA hairpin termini in various tissues in mice. J Virol (2007) 1.35
Therapeutic application of RNAi: is mRNA targeting finally ready for prime time? J Clin Invest (2007) 1.34
Capped small RNAs and MOV10 in human hepatitis delta virus replication. Nat Struct Mol Biol (2008) 1.34
In vivo antiviral efficacy of prenylation inhibitors against hepatitis delta virus. J Clin Invest (2003) 1.33
Silencing of hepatic fatty acid transporter protein 5 in vivo reverses diet-induced non-alcoholic fatty liver disease and improves hyperglycemia. J Biol Chem (2008) 1.32
The host response to adenovirus, helper-dependent adenovirus, and adeno-associated virus in mouse liver. Mol Ther (2008) 1.31
Generation of adult human induced pluripotent stem cells using nonviral minicircle DNA vectors. Nat Protoc (2010) 1.30
Thermodynamic stability of small hairpin RNAs highly influences the loading process of different mammalian Argonautes. Proc Natl Acad Sci U S A (2011) 1.28
FATP2 is a hepatic fatty acid transporter and peroxisomal very long-chain acyl-CoA synthetase. Am J Physiol Endocrinol Metab (2010) 1.26
Molecular analysis of chromosomal rearrangements in mammalian cells after phiC31-mediated integration. Hum Gene Ther (2006) 1.25
Minicircle DNA vectors achieve sustained expression reflected by active chromatin and transcriptional level. Mol Ther (2012) 1.24
Comparison of adenoviral and adeno-associated viral vectors for pancreatic gene delivery in vivo. Hum Gene Ther (2004) 1.22
A two-hybrid screen identifies cathepsins B and L as uncoating factors for adeno-associated virus 2 and 8. Mol Ther (2007) 1.19
A new adenoviral helper-dependent vector results in long-term therapeutic levels of human coagulation factor IX at low doses in vivo. Blood (2002) 1.18
How do miRNAs mediate translational repression? Silence (2010) 1.17
Postintegrative gene silencing within the Sleeping Beauty transposition system. Mol Cell Biol (2007) 1.15
Adeno-associated virus vectors for short hairpin RNA expression. Methods Enzymol (2005) 1.14
Double knockdown of prolyl hydroxylase and factor-inhibiting hypoxia-inducible factor with nonviral minicircle gene therapy enhances stem cell mobilization and angiogenesis after myocardial infarction. Circulation (2011) 1.14
Liver tissue engineering at extrahepatic sites in mice as a potential new therapy for genetic liver diseases. Hepatology (2005) 1.14
A prenylation inhibitor prevents production of infectious hepatitis delta virus particles. J Virol (2002) 1.13
RNAi and gene therapy: a mutual attraction. Hematology Am Soc Hematol Educ Program (2007) 1.10
Low-level shRNA cytotoxicity can contribute to MYC-induced hepatocellular carcinoma in adult mice. Mol Ther (2009) 1.09
Minicircle DNA-based gene therapy coupled with immune modulation permits long-term expression of α-L-iduronidase in mice with mucopolysaccharidosis type I. Mol Ther (2010) 1.08
Host factors that impact the biodistribution and persistence of multipotent adult progenitor cells. Blood (2006) 1.08
Helper-independent and AAV-ITR-independent chromosomal integration of double-stranded linear DNA vectors in mice. Mol Ther (2003) 1.08
Ribosomal DNA integrating rAAV-rDNA vectors allow for stable transgene expression. Mol Ther (2012) 1.08
Episomal persistence of recombinant adenoviral vector genomes during the cell cycle in vivo. J Virol (2003) 1.06
A mini-intronic plasmid (MIP): a novel robust transgene expression vector in vivo and in vitro. Mol Ther (2013) 1.05
A direct comparison of two nonviral gene therapy vectors for somatic integration: in vivo evaluation of the bacteriophage integrase phiC31 and the Sleeping Beauty transposase. Mol Ther (2005) 1.04
Radioprotection in vitro and in vivo by minicircle plasmid carrying the human manganese superoxide dismutase transgene. Hum Gene Ther (2008) 1.04
Characterization of the relationship of AAV capsid domain swapping to liver transduction efficiency. Mol Ther (2007) 1.02
Pathways of removal of free DNA vector ends in normal and DNA-PKcs-deficient SCID mouse hepatocytes transduced with rAAV vectors. Hum Gene Ther (2003) 1.02
Looking into the safety of AAV vectors. Nature (2003) 1.02
Increased maintenance and persistence of transgenes by excision of expression cassettes from plasmid sequences in vivo. Hum Gene Ther (2005) 1.01
A gene-deleted adenoviral vector results in phenotypic correction of canine hemophilia B without liver toxicity or thrombocytopenia. Blood (2003) 1.00
Hyperactive sleeping beauty transposase enables persistent phenotypic correction in mice and a canine model for hemophilia B. Mol Ther (2010) 1.00
The extragenic spacer length between the 5' and 3' ends of the transgene expression cassette affects transgene silencing from plasmid-based vectors. Mol Ther (2012) 0.98
A rapid protocol for construction and production of high-capacity adenoviral vectors. Nat Protoc (2009) 0.97
Slicing-independent RISC activation requires the argonaute PAZ domain. Curr Biol (2012) 0.96
Combined proteomic-RNAi screen for host factors involved in human hepatitis delta virus replication. RNA (2009) 0.95
Free DNA ends are essential for concatemerization of synthetic double-stranded adeno-associated virus vector genomes transfected into mouse hepatocytes in vivo. Mol Ther (2003) 0.95
Translational data from adeno-associated virus-mediated gene therapy of hemophilia B in dogs. Hum Gene Ther Clin Dev (2015) 0.95
Expression determinants of mammalian argonaute proteins in mediating gene silencing. Nucleic Acids Res (2011) 0.95
The effect of age on hepatic gene transfer with self-inactivating lentiviral vectors in vivo. Mol Ther (2003) 0.94
AAV vectors containing rDNA homology display increased chromosomal integration and transgene persistence. Mol Ther (2012) 0.93
Somatic integration from an adenoviral hybrid vector into a hot spot in mouse liver results in persistent transgene expression levels in vivo. Mol Ther (2007) 0.93
Optimization of cis-acting elements for gene expression from nonviral vectors in vivo. Hum Gene Ther (2003) 0.92
Advancing molecular therapies through in vivo bioluminescent imaging. Mol Imaging (2003) 0.92
Gene transfer to mouse heart and skeletal muscles using a minicircle expressing human vascular endothelial growth factor. J Cardiovasc Pharmacol (2009) 0.91