Published in Mol Ther on October 12, 2005
Hematopoietic stem-cell gene therapy of hemophilia A incorporating a porcine factor VIII transgene and nonmyeloablative conditioning regimens. Blood (2007) 1.28
Directed engineering of a high-expression chimeric transgene as a strategy for gene therapy of hemophilia A. Mol Ther (2009) 1.19
Progress toward inducing immunologic tolerance to factor VIII. Blood (2013) 1.10
Immunomodulation of transgene responses following naked DNA transfer of human factor VIII into hemophilia A mice. Blood (2006) 1.08
Predicting preferential DNA vector insertion sites: implications for functional genomics and gene therapy. Genome Biol (2007) 1.08
Animal models of hemophilia. Prog Mol Biol Transl Sci (2012) 1.04
Anti-CD3 antibodies modulate anti-factor VIII immune responses in hemophilia A mice after factor VIII plasmid-mediated gene therapy. Blood (2009) 1.03
Comparison of factor VIII transgenes bioengineered for improved expression in gene therapy of hemophilia A. Hum Gene Ther (2009) 0.99
In vivo expansion of regulatory T cells with IL-2/IL-2 mAb complexes prevents anti-factor VIII immune responses in hemophilia A mice treated with factor VIII plasmid-mediated gene therapy. Mol Ther (2011) 0.94
Intraosseous delivery of lentiviral vectors targeting factor VIII expression in platelets corrects murine hemophilia A. Mol Ther (2015) 0.93
Genetic modification of stem cells for transplantation. Adv Drug Deliv Rev (2007) 0.93
Correction of murine ADAMTS13 deficiency by hematopoietic progenitor cell-mediated gene therapy. Blood (2009) 0.92
Erythroid-specific human factor IX delivery from in vivo selected hematopoietic stem cells following nonmyeloablative conditioning in hemophilia B mice. Mol Ther (2008) 0.87
Stable gammaretroviral vector expression during embryonic stem cell-derived in vitro hematopoietic development. Mol Ther (2006) 0.87
Gene therapy for haemophilia: prospects and challenges to prevent or reverse inhibitor formation. Br J Haematol (2011) 0.86
Immunomodulation for inhibitors in hemophilia A: the important role of Treg cells. Expert Rev Hematol (2010) 0.85
In vivo enrichment of genetically manipulated platelets corrects the murine hemophilic phenotype and induces immune tolerance even using a low multiplicity of infection. J Thromb Haemost (2014) 0.84
Advancements in gene transfer-based therapy for hemophilia A. Expert Rev Hematol (2009) 0.83
Extrahepatic sources of factor VIII potentially contribute to the coagulation cascade correcting the bleeding phenotype of mice with hemophilia A. Haematologica (2015) 0.82
Factor VIII delivered by haematopoietic stem cell-derived B cells corrects the phenotype of haemophilia A mice. Thromb Haemost (2011) 0.82
Transgene expression levels determine the immunogenicity of transduced hematopoietic grafts in partially myeloablated mice. Mol Ther (2009) 0.82
Advances in Overcoming Immune Responses following Hemophilia Gene Therapy. J Genet Syndr Gene Ther (2011) 0.81
Progress and challenges in the development of a cell-based therapy for hemophilia A. J Thromb Haemost (2014) 0.81
Gene-delivery systems for iPS cell generation. Expert Opin Biol Ther (2010) 0.79
Supplying clotting factors from hematopoietic stem cell-derived erythroid and megakaryocytic lineage cells. Mol Ther (2009) 0.79
Delivery of nucleic acid therapeutics by genetically engineered hematopoietic stem cells. Adv Drug Deliv Rev (2010) 0.79
Hematopoietic stem cells. Methods Enzymol (2006) 0.78
Engineered Hematopoietic Stem Cells as Therapeutics for Hemophilia A. J Genet Syndr Gene Ther (2011) 0.77
Treatment of Hemophilia A in Utero and Postnatally using Sheep as a Model for Cell and Gene Delivery. J Genet Syndr Gene Ther (2012) 0.77
Delivery of factor VIII gene into skeletal muscle cells using lentiviral vector. Yonsei Med J (2009) 0.76
In utero stem cell transplantation and gene therapy: rationale, history, and recent advances toward clinical application. Mol Ther Methods Clin Dev (2016) 0.76
Effects of FVIII immunity on hepatocyte and hematopoietic stem cell-directed gene therapy of murine hemophilia A. Mol Ther Methods Clin Dev (2016) 0.76
Hemophilia A: an ideal disease to correct in utero. Front Pharmacol (2014) 0.75
Induction of Hematopoietic Microchimerism by Gene-Modified BMT Elicits Antigen-Specific B and T Cell Unresponsiveness toward Gene Therapy Products. Front Immunol (2016) 0.75
Immune tolerance induced by platelet-targeted Factor VIII gene therapy in hemophilia A mice is CD4 T cell-mediated. J Thromb Haemost (2017) 0.75
Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science (2002) 6.46
Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A. Nat Genet (1995) 3.74
Clonal dominance of hematopoietic stem cells triggered by retroviral gene marking. Science (2005) 3.58
The enemy within: keeping self-reactive T cells at bay in the periphery. Nat Rev Immunol (2002) 3.47
Haemophilias A and B. Lancet (2003) 2.68
Targeted expression of major histocompatibility complex (MHC) class II molecules demonstrates that dendritic cells can induce negative but not positive selection of thymocytes in vivo. J Exp Med (1997) 2.33
Proceedings: A more uniform measurement of factor VIII inhibitors. Thromb Diath Haemorrh (1975) 2.10
Back to central tolerance. Immunity (2004) 1.88
CD8 T cell ignorance or tolerance to islet antigens depends on antigen dose. Proc Natl Acad Sci U S A (1999) 1.55
Biochemical, immunological, and in vivo functional characterization of B-domain-deleted factor VIII. Blood (1993) 1.53
Prevention and treatment of factor VIII inhibitors in murine hemophilia A. Blood (2000) 1.36
Nonmyeloablative conditioning is sufficient to allow engraftment of EGFP-expressing bone marrow and subsequent acceptance of EGFP-transgenic skin grafts in mice. Blood (2003) 1.33
Inhibitor antibody development and T cell response to human factor VIII in murine hemophilia A. Thromb Haemost (1999) 1.30
Efficient production of human FVIII in hemophilic mice using lentiviral vectors. Mol Ther (2003) 1.29
Naked DNA transfer of Factor VIII induced transgene-specific, species-independent immune response in hemophilia A mice. Mol Ther (2004) 1.24
Anti-factor VIII antibodies of hemophiliac patients are frequently directed towards nonfunctional determinants and do not exhibit isotypic restriction. Blood (1993) 1.19
Mechanisms of tolerance induction by a gene-transferred peptide-IgG fusion protein expressed in B lineage cells. J Immunol (2000) 1.17
Inhibitors in hemophilia A: mechanisms of inhibition, management and perspectives. Blood Coagul Fibrinolysis (2004) 1.17
Introduction of a xenogeneic gene via hematopoietic stem cells leads to specific tolerance in a rhesus monkey model. Mol Ther (2000) 1.14
Sustained phenotypic correction of hemophilia a mice following oncoretroviral-mediated expression of a bioengineered human factor VIII gene in long-term hematopoietic repopulating cells. Mol Ther (2004) 1.12
Mechanism of the immune response to human factor VIII in murine hemophilia A. Thromb Haemost (2001) 1.10
Genetic induction of immune tolerance to human clotting factor VIII in a mouse model for hemophilia A. Proc Natl Acad Sci U S A (1998) 1.07
Tolerance in mixed chimerism - a role for regulatory cells? Trends Immunol (2004) 1.06
Induction of transgene-specific immunological tolerance in myeloablated nonhuman primates using lentivirally transduced CD34+ progenitor cells. Mol Ther (2003) 1.04
Engraftment of retroviral EGFP-transduced bone marrow in mice prevents rejection of EGFP-transgenic skin grafts. Mol Ther (2003) 1.03
Induction of central tolerance by mature T cells. J Immunol (2004) 0.99
Progress toward vector design for hematopoietic stem cell gene therapy. Curr Gene Ther (2001) 0.97
Gene transfer as an approach to treating hemophilia. Semin Thromb Hemost (2003) 0.95
Bypass of senescence, immortalization, and transformation of human hematopoietic progenitor cells. Stem Cells (2005) 0.95
Prevention of autoimmune disease by retroviral-mediated gene therapy. J Immunol (1995) 0.93
Induction of B-cell tolerance by retroviral gene therapy. Blood (2000) 0.92
Toward gene therapy in haemophilia A: retrovirus-mediated transfer of a factor VIII gene into murine haematopoietic progenitor cells. Thromb Haemost (1992) 0.91
Noncoagulation inhibitory factor VIII antibodies after induction of tolerance to factor VIII in hemophilia A patients. Blood (1990) 0.91
Development of improved factor VIII molecules and new gene transfer approaches for hemophilia A. Curr Gene Ther (2003) 0.90
Specific prolongation of skin graft survival following retroviral transduction of bone marrow with an allogeneic major histocompatibility complex gene. Transplantation (1993) 0.89
Expression of antigen on mature lymphocytes is required to induce T cell tolerance by gene therapy. J Immunol (2002) 0.88
Establishing immunological tolerance through the induction of molecular chimerism. Front Biosci (2002) 0.87
Induction of donor-specific tolerance in sublethally irradiated recipients by gene therapy. Mol Ther (2005) 0.83
Use of gene therapy to suppress the antigen-specific immune responses in mice to an HLA antigen. Transplantation (1996) 0.81
Cardiac allograft acceptance after localized bone marrow transplantation by isolated limb perfusion in nonmyeloablated recipients. Stem Cells (2003) 0.78
Interferon-γ links ultraviolet radiation to melanomagenesis in mice. Nature (2011) 3.56
Transfer of a TCR gene derived from a patient with a marked antitumor response conveys highly active T-cell effector functions. Hum Gene Ther (2005) 3.49
High-level sustained transgene expression in human embryonic stem cells using lentiviral vectors. Stem Cells (2003) 2.42
Reduced lymphomyeloid repopulating activity from adult bone marrow and fetal liver of mice lacking expression of STAT5. Blood (2002) 1.96
HOX and non-HOX homeobox genes in leukemic hematopoiesis. Stem Cells (2002) 1.94
Functional analysis of various promoters in lentiviral vectors at different stages of in vitro differentiation of mouse embryonic stem cells. Mol Ther (2007) 1.76
Optimized flow cytometric analysis of central nervous system tissue reveals novel functional relationships among cells expressing CD133, CD15, and CD24. Stem Cells (2007) 1.68
Does retroviral insertional mutagenesis play a role in the generation of induced pluripotent stem cells? Mol Ther (2008) 1.47
Performance- and safety-enhanced lentiviral vectors containing the human interferon-beta scaffold attachment region and the chicken beta-globin insulator. Blood (2003) 1.40
Catalytic-dependent and -independent roles of SHP-2 tyrosine phosphatase in interleukin-3 signaling. Oncogene (2003) 1.28
Cell intrinsic defects in cytokine responsiveness of STAT5-deficient hematopoietic stem cells. Blood (2002) 1.25
Sustained phenotypic correction of hemophilia a mice following oncoretroviral-mediated expression of a bioengineered human factor VIII gene in long-term hematopoietic repopulating cells. Mol Ther (2004) 1.12
Role of the docking protein Gab2 in beta(1)-integrin signaling pathway-mediated hematopoietic cell adhesion and migration. Blood (2002) 1.00
Hematopoietic cell fate and the initiation of leukemic properties in primitive primary human cells are influenced by Ras activity and farnesyltransferase inhibition. Mol Cell Biol (2004) 1.00
G1/S transcriptional networks modulated by the HOX11/TLX1 oncogene of T-cell acute lymphoblastic leukemia. Oncogene (2005) 0.98
Flow cytometry of fluorescent proteins. Methods (2012) 0.97
TLX1/HOX11-mediated disruption of primary thymocyte differentiation prior to the CD4+CD8+ double-positive stage. Br J Haematol (2006) 0.96
Identification of an ABCB1 (P-glycoprotein)-positive carfilzomib-resistant myeloma subpopulation by the pluripotent stem cell fluorescent dye CDy1. Am J Hematol (2013) 0.96
BCL-2 and BCL-XL restrict lineage choice during hematopoietic differentiation. J Biol Chem (2003) 0.96
Multiparameter flow cytometry of fluorescent protein reporters. Methods Mol Biol (2004) 0.95
Acetylation-dependent oncogenic activity of metastasis-associated protein 1 co-regulator. EMBO Rep (2010) 0.95
Bypass of senescence, immortalization, and transformation of human hematopoietic progenitor cells. Stem Cells (2005) 0.95
Specific homeodomain-DNA interactions are required for HOX11-mediated transformation. Blood (2003) 0.93
Overexpression of rab7 enhances the kinetics of antigen processing and presentation with MHC class II molecules in B cells. Int Immunol (2002) 0.92
Development of improved factor VIII molecules and new gene transfer approaches for hemophilia A. Curr Gene Ther (2003) 0.90
Overview of the HIV-1 Lentiviral Vector System. Curr Protoc Mol Biol (2002) 0.88
Role of TLX1 in T-cell acute lymphoblastic leukaemia pathogenesis. Br J Haematol (2009) 0.88
Generation of HIV-1-based lentiviral vector particles. Curr Protoc Mol Biol (2002) 0.88
Phenotype correction of Fanconi anemia group A hematopoietic stem cells using lentiviral vector. Mol Ther (2003) 0.88
Stable gammaretroviral vector expression during embryonic stem cell-derived in vitro hematopoietic development. Mol Ther (2006) 0.87
Immortalization of yolk sac-derived precursor cells. Blood (2002) 0.87
TLX1 and NOTCH coregulate transcription in T cell acute lymphoblastic leukemia cells. Mol Cancer (2010) 0.85
Resistance to fas-induced apoptosis in cells from human atherosclerotic lesions: elevated Bcl-XL inhibits apoptosis and caspase activation. J Vasc Res (2007) 0.85
Transcriptional activation by TLX1/HOX11 involves Gro/TLE corepressors. Biochem Biophys Res Commun (2009) 0.84
Reducing the genotoxic potential of retroviral vectors. Methods Mol Biol (2008) 0.84
Factor VIII delivered by haematopoietic stem cell-derived B cells corrects the phenotype of haemophilia A mice. Thromb Haemost (2011) 0.82
Genetic analysis of the ATG7 gene promoter in sporadic Parkinson's disease. Neurosci Lett (2013) 0.82
Detection and enrichment of hematopoietic stem cells by side population phenotype. Methods Mol Biol (2004) 0.81
Analysis of violet-excited fluorochromes by flow cytometry using a violet laser diode. Cytometry A (2003) 0.81
Retroviral transduction in fetal thymic organ culture. Methods Mol Med (2005) 0.80
A novel and functional variant within the ATG5 gene promoter in sporadic Parkinson's disease. Neurosci Lett (2013) 0.80
Integrative molecular and developmental biology of adult stem cells. Biol Cell (2003) 0.80
SCID repopulating cells derived from unmobilised adult human peripheral blood. Curr Med Res Opin (2004) 0.79
Specific pharmacological dimerization of KDR in lentivirally transduced human hematopoietic cells activates anti-apoptotic and proliferative mechanisms. FASEB J (2005) 0.79
TLX1 (HOX11) immortalization of embryonic stem cell-derived and primary murine hematopoietic progenitors. Curr Protoc Stem Cell Biol (2008) 0.78
Hematopoietic stem cells. Methods Enzymol (2006) 0.78
Lentiviral fluorescent protein expression vectors for biotinylation proteomics. Methods Mol Biol (2011) 0.77
Strategies to insulate lentiviral vector-expressed transgenes. Methods Mol Biol (2010) 0.77
Hematopoietic immortalizing function of the NKL-subclass homeobox gene TLX1. Genes Chromosomes Cancer (2010) 0.77
Tissue inhibitor of matrix metalloproteinase-1 overexpression in M1 myeloblasts impairs IL-6-induced differentiation. Oncogene (2004) 0.77
Apoptotic role of IKK in T-ALL therapeutic response. Mol Cancer Res (2011) 0.76
Human immunodeficiency virus type 1-based vectors for gene delivery to human hematopoietic stem cells. Methods Mol Med (2003) 0.76
New feature: stem cells in the news. Stem Cells (2002) 0.75
Identification of a novel 21bp-insertion variant within the LC3B gene promoter in sporadic Parkinson's disease. Transl Res (2013) 0.75
Stem cell bouillabaisse-potpourri. Stem Cells (2002) 0.75
Advancing the fast-paced field of stem cell research: stem cells increases from 6 to 10 issues in its 23rd year of publication. Stem Cells (2005) 0.75
Somatic stem cell plasticity: to be or not to be. Stem Cells (2002) 0.75
The tao of hematopoietic stem cells: toward a unified theory of tissue regeneration. ScientificWorldJournal (2002) 0.75
Editorial retraction. Stem Cells (2006) 0.75