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Molecular evolution of a novel hyperactive Sleeping Beauty transposase enables robust stable gene transfer in vertebrates

Nature genetics, 2009-06, Vol.41 (6), p.753-761

2009

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Transposon-mediated gene transfer into adult and induced pluripotent stem cells

Current gene therapy, 2011-10, Vol.11 (5), p.406-413

2011

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Restoration of plasma von Willebrand factor deficiency is sufficient to correct thrombus formation after gene therapy for severe von Willebrand disease

Arteriosclerosis, thrombosis, and vascular biology, 2008-09, Vol.28 (9), p.1621-1626

2008

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Efficient Lentiviral Transduction and Improved Engraftment of Human Bone Marrow Mesenchymal Cells

Stem cells (Dayton, Ohio), 2006-04, Vol.24 (4), p.896-907

2006

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Xenogeneic liver models for gene therapy

Human gene therapy, 2010-01, Vol.21 (1), p.1-4

2010

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Emerging potential of transposons for gene therapy and generation of induced pluripotent stem cells

Blood, 2009-08, Vol.114 (8), p.1461-1468

2009

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Codon optimization of human factor VIII cDNAs leads to high-level expression

Blood, 2011-01, Vol.117 (3), p.798-807

2011

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Comparative Analysis of Transposable Element Vector Systems in Human Cells

Molecular therapy, 2010-06, Vol.18 (6), p.1200-1209

2010

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Intrinsic cell memory reinforces myogenic commitment of pericyte-derived iPSCs

The Journal of pathology, 2011-04, Vol.223 (5), p.593-603

2011

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Efficient stable gene transfer into human cells by the Sleeping Beauty transposon vectors

Methods (San Diego, Calif.), 2009-11, Vol.49 (3), p.287-297

2009

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Nanoparticles for the delivery of genes and drugs to human hepatocytes

Nature biotechnology, 2003-08, Vol.21 (8), p.885-890

2003

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Onco-Retroviral and Lentiviral Vector-Based Gene Therapy for Hemophilia: Preclinical Studies

Seminars in thrombosis and hemostasis, 2004-04, Vol.30 (2), p.185-195

2004

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Lentiviral vectors containing the human immunodeficiency virus type-1 central polypurine tract can efficiently transduce nondividing hepatocytes and antigen-presenting cells in vivo

Blood, 2002-08, Vol.100 (3), p.813-822

2002

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Efficacy and safety of adeno‐associated viral vectors based on serotype 8 and 9 vs. lentiviral vectors for hemophilia B gene therapy

Journal of thrombosis and haemostasis, 2007-01, Vol.5 (1), p.16-24

2007

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Novel Hyperactive Transposons for Genetic Modification of Induced Pluripotent and Adult Stem Cells: A Nonviral Paradigm for Coaxed Differentiation

Stem cells (Dayton, Ohio), 2010-10, Vol.28 (10), p.1760-1771

2010

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Preclinical and clinical progress in hemophilia gene therapy

Current opinion in hematology, 2010-09, Vol.17 (5), p.387-392

2010

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Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors

Blood, 2003-03, Vol.101 (5), p.1734-1743

2003

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Moving gene therapy forward with mobile DNA

Human gene therapy, 2009-12, Vol.20 (12), p.1559-1561

2009

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Gene therapy for hemophilia: hopes and hurdles

Critical reviews in oncology/hematology, 1998-09, Vol.28 (3), p.153-171

1998

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PiggyBac toolbox

Methods in molecular biology (Clifton, N.J.), 2012, Vol.859, p.241

2012

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Clinical Gene Transfer Studies for Hemophilia A

Seminars in thrombosis and hemostasis, 2004-04, Vol.30 (2), p.249-256

2004

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Long-Term Expression of Human Coagulation Factor VIII and Correction of Hemophilia A after in vivo Retroviral Gene Transfer in Factor VIII-Deficient Mice

Proceedings of the National Academy of Sciences - PNAS, 1999-08, Vol.96 (18), p.10379-10384

1999

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Bone marrow stromal cells as targets for gene therapy

Current gene therapy, 2002-05, Vol.2 (2), p.195-209

2002

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Biosafety of adenoviral vectors

Current gene therapy, 2003-12, Vol.3 (6), p.527-543

2003

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Long-term persistence of human bone marrow stromal cells transduced with factor VIII-retroviral vectors and transient production of therapeutic levels of human factor VIII in nonmyeloablated immunodeficient mice

Human gene therapy, 2000-03, Vol.11 (5), p.729-738

2000

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Molecular evolution of a novel hyperactive Sleeping Beauty transposase enables robust stable gene transfer in vertebrates

Transposon-mediated gene transfer into adult and induced pluripotent stem cells

Restoration of plasma von Willebrand factor deficiency is sufficient to correct thrombus formation after gene therapy for severe von Willebrand disease

Efficient Lentiviral Transduction and Improved Engraftment of Human Bone Marrow Mesenchymal Cells

Xenogeneic liver models for gene therapy

Emerging potential of transposons for gene therapy and generation of induced pluripotent stem cells

Codon optimization of human factor VIII cDNAs leads to high-level expression

Comparative Analysis of Transposable Element Vector Systems in Human Cells

Intrinsic cell memory reinforces myogenic commitment of pericyte-derived iPSCs

Efficient stable gene transfer into human cells by the Sleeping Beauty transposon vectors

Nanoparticles for the delivery of genes and drugs to human hepatocytes

Onco-Retroviral and Lentiviral Vector-Based Gene Therapy for Hemophilia: Preclinical Studies

Lentiviral vectors containing the human immunodeficiency virus type-1 central polypurine tract can efficiently transduce nondividing hepatocytes and antigen-presenting cells in vivo

Efficacy and safety of adeno‐associated viral vectors based on serotype 8 and 9 vs. lentiviral vectors for hemophilia B gene therapy

Novel Hyperactive Transposons for Genetic Modification of Induced Pluripotent and Adult Stem Cells: A Nonviral Paradigm for Coaxed Differentiation

Preclinical and clinical progress in hemophilia gene therapy

Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors

Moving gene therapy forward with mobile DNA

Gene therapy for hemophilia: hopes and hurdles

PiggyBac toolbox

Clinical Gene Transfer Studies for Hemophilia A

Long-Term Expression of Human Coagulation Factor VIII and Correction of Hemophilia A after in vivo Retroviral Gene Transfer in Factor VIII-Deficient Mice

Bone marrow stromal cells as targets for gene therapy

Biosafety of adenoviral vectors

Long-term persistence of human bone marrow stromal cells transduced with factor VIII-retroviral vectors and transient production of therapeutic levels of human factor VIII in nonmyeloablated immunodeficient mice

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