Published in PLoS One on August 15, 2014
Precise correction of the dystrophin gene in duchenne muscular dystrophy patient induced pluripotent stem cells by TALEN and CRISPR-Cas9. Stem Cell Reports (2014) 1.88
piggyBac-ing models and new therapeutic strategies. Trends Biotechnol (2015) 0.89
The Utilization of Rehabilitation in Patients with Hemophilia A in Taiwan: A Nationwide Population-Based Study. PLoS One (2016) 0.78
In situ genetic correction of F8 intron 22 inversion in hemophilia A patient-specific iPSCs. Sci Rep (2016) 0.77
Donor antigen-primed regulatory T cells permit liver regeneration and phenotype correction in hemophilia A mouse by allogeneic bone marrow stem cells. Stem Cell Res Ther (2015) 0.77
A genome editing primer for the hematologist. Blood (2016) 0.77
Proteasome impairment in neural cells derived from HMSN-P patient iPSCs. Mol Brain (2017) 0.75
Calcium dysregulation contributes to neurodegeneration in FTLD patient iPSC-derived neurons. Sci Rep (2016) 0.75
Preclinical and clinical advances in transposon-based gene therapy. Biosci Rep (2017) 0.75
Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell (2007) 101.42
Efficient selection for high-expression transfectants with a novel eukaryotic vector. Gene (1991) 38.64
LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science (2003) 19.77
Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med (2011) 11.01
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 complementary transposon tool kit for Drosophila melanogaster using P and piggyBac. Nat Genet (2004) 8.55
Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA. Gene Ther (1999) 8.28
Efficient transposition of the piggyBac (PB) transposon in mammalian cells and mice. Cell (2005) 7.29
A fresh look at iPS cells. Cell (2009) 5.73
In vivo genome editing restores haemostasis in a mouse model of haemophilia. Nature (2011) 4.81
PiggyBac transposon-mediated gene transfer in human cells. Mol Ther (2007) 4.07
A hyperactive piggyBac transposase for mammalian applications. Proc Natl Acad Sci U S A (2011) 2.68
Isolation of human iPS cells using EOS lentiviral vectors to select for pluripotency. Nat Methods (2009) 2.51
Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype. Blood (2003) 2.21
Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs. Blood (2006) 2.03
Acute direct adenoviral vector cytotoxicity and chronic, but not acute, inflammatory responses correlate with decreased vector-mediated transgene expression in the brain. Mol Ther (2001) 1.55
Mobilization of giant piggyBac transposons in the mouse genome. Nucleic Acids Res (2011) 1.49
Phase 1 trial of FVIII gene transfer for severe hemophilia A using a retroviral construct administered by peripheral intravenous infusion. Blood (2003) 1.47
Computer-assisted hydrodynamic gene delivery. Mol Ther (2008) 1.40
Gene transfer efficiency and genome-wide integration profiling of Sleeping Beauty, Tol2, and piggyBac transposons in human primary T cells. Mol Ther (2010) 1.38
piggyBac transposon-mediated long-term gene expression in mice. Mol Ther (2010) 1.33
Efficient production of human FVIII in hemophilic mice using lentiviral vectors. Mol Ther (2003) 1.29
Ex vivo gene therapy for hemophilia A that enhances safe delivery and sustained in vivo factor VIII expression from lentivirally engineered endothelial progenitors. Stem Cells (2007) 1.22
Hyperactive piggyBac gene transfer in human cells and in vivo. Hum Gene Ther (2011) 1.21
Image-guided, lobe-specific hydrodynamic gene delivery to swine liver. Mol Ther (2009) 1.20
Functional roles of the factor VIII B domain. Haemophilia (2009) 1.16
Persistent expression of factor VIII in vivo following nonprimate lentiviral gene transfer. Blood (2005) 1.14
Uncovering and dissecting the genotoxicity of self-inactivating lentiviral vectors in vivo. Mol Ther (2014) 1.13
Complete correction of hemophilia A with adeno-associated viral vectors containing a full-size expression cassette. Hum Gene Ther (2008) 1.13
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
Neonatal helper-dependent adenoviral vector gene therapy mediates correction of hemophilia A and tolerance to human factor VIII. Proc Natl Acad Sci U S A (2011) 1.11
Efficacy and safety of Sleeping Beauty transposon-mediated gene transfer in preclinical animal studies. Curr Gene Ther (2011) 1.08
Novel forms of B-domain-deleted recombinant factor VIII molecules. Construction and biochemical characterization. Eur J Biochem (1995) 1.06
Antigenicity of putative phospholipid membrane-binding residues in factor VIII. Blood (2001) 0.90
Preclinical and clinical progress in hemophilia gene therapy. Curr Opin Hematol (2010) 0.87
Gene therapy strategies for hemophilia: benefits versus risks. J Gene Med (2010) 0.85
Nanotechnology for therapy and imaging of liver diseases. J Hepatol (2011) 0.84
Long-term expression of human coagulation factor VIII in a tolerant mouse model using the φC31 integrase system. Hum Gene Ther (2012) 0.84
Gene therapy for haemophilia...yes, but...with non-viral vectors? Haemophilia (2009) 0.83
Abnormal factor VIII Hiroshima: defect in crucial proteolytic cleavage by thrombin at Arg1689 detected by a novel ELISA. Br J Haematol (1994) 0.79
A novel cell-sheet technology that achieves durable factor VIII delivery in a mouse model of hemophilia A. PLoS One (2013) 0.79