Search Results - "Mulligan, R C"
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Lack of toxicity of alpha-sarcoglycan overexpression supports clinical gene transfer trial in LGMD2D
Published in Neurology (22-07-2008)“…Alpha-sarcoglycan (alpha-SG) deficiency (limb-girdle muscular dystrophy [LGMD] type 2D) is the most common form of sarcoglycan-LGMD. No treatment is currently…”
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2
Dystrophin expression in the mdx mouse restored by stem cell transplantation
Published in Nature (London) (23-09-1999)“…The development of cell or gene therapies for diseases involving cells that are widely distributed throughout the body has been severely hampered by the…”
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3
Vaccination with Irradiated Tumor Cells Engineered to Secrete Murine Granulocyte-Macrophage Colony-Stimulating Factor Stimulates Potent, Specific, and Long-Lasting Anti-Tumor Immunity
Published in Proceedings of the National Academy of Sciences - PNAS (15-04-1993)“…To compare the ability of different cytokines and other molecules to enhance the immunogenicity of tumor cells, we generated 10 retroviruses encoding potential…”
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4
Lethal skeletal dysplasia from targeted disruption of the parathyroid hormone-related peptide gene
Published in Genes & development (01-02-1994)“…The parathyroid hormone-related peptide (PTHrP) gene was disrupted in murine embryonic stem cells by homologous recombination, and the null allele was…”
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5
A Stable Human-Derived Packaging Cell Line for Production of High Titer Retrovirus/Vesicular Stomatitis Virus G Pseudotypes
Published in Proceedings of the National Academy of Sciences - PNAS (15-10-1996)“…We have generated a human 293-derived retroviral packaging cell line (293GPG) capable of producing high titers of recombinant Moloney murine leukemia virus…”
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6
Role for interleukin-3 in mast-cell and basophil development and in immunity to parasites
Published in Nature (London) (05-03-1998)“…The cytokine interleukin-3 (IL-3), which can be derived from T cells and other sources, is a potentially important link between the immune and haematopoietic…”
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7
Plasmid DNA encapsulation and release from solvent diffusion nanospheres
Published in Journal of controlled release (29-01-2001)“…The first step toward hydrophobic polymer-based nanospheres for gene delivery is to encapsulate and release plasmid DNA. However, encapsulating large…”
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Safe and Efficient Generation of Recombinant Retroviruses with Amphotropic and Ecotropic Host Ranges
Published in Proceedings of the National Academy of Sciences - PNAS (01-09-1988)“…We have constructed a set of packaging cell lines useful for the generation of helper-free recombinant retroviruses with amphotropic and ecotropic host ranges…”
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9
PTH/PTHrP Receptor in Early Development and Indian Hedgehog-Regulated Bone Growth
Published in Science (American Association for the Advancement of Science) (02-08-1996)“…The PTH/PTHrP receptor binds to two ligands with distinct functions: the calcium-regulating hormone, parathyroid hormone (PTH), and the paracrine factor,…”
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10
Heterogeneity of the Angiogenic Response Induced in Different Normal Adult Tissues by Vascular Permeability Factor/Vascular Endothelial Growth Factor
Published in Laboratory investigation (01-01-2000)“…Vascular permeability factor/vascular endothelial growth factor (VPF/VEGF) is an angiogenic cytokine with potential for the treatment of tissue ischemia. To…”
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11
Effects of Retroviral Vector Design on Expression of Human Adenosine Deaminase in Murine Bone Marrow Transplant Recipients Engrafted with Genetically Modified Cells
Published in Proceedings of the National Academy of Sciences - PNAS (18-07-1995)“…To determine which features of retroviral vector design most critically affect gene expression in hematopoietic cells in vivo, we have constructed a variety of…”
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12
Surfactant metabolism in transgenic mice after granulocyte macrophage-colony stimulating factor ablation
Published in The American journal of physiology (01-04-1996)“…Mice made granulocyte macrophage-colony stimulating factor (GM-CSF)-deficient by homologous recombination maintain normal steady-state hematopoiesis but have…”
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13
Involvement of Granulocyte-Macrophage Colony-Stimulating Factor in Pulmonary Homeostasis
Published in Science (American Association for the Advancement of Science) (29-04-1994)“…The in vivo function of murine granulocyte-macrophage colony-stimulating factor (GM-CSF) was investigated in mice, carrying a null allele of the GM-CSF gene,…”
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14
Gene therapy to target dendritic cells from blood to lymph nodes
Published in Gene therapy (01-08-2003)“…Peripheral lymph nodes (PLN) are strategic microenvironments where antigen-presenting dendritic cells (DC), loaded with environmental antigens, and naive…”
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15
Pulmonary epithelial cell expression of GM-CSF corrects the alveolar proteinosis in GM-CSF-deficient mice
Published in The Journal of clinical investigation (01-02-1996)“…Mutation of the granulocyte-macrophage colony-stimulating factor (GM-CSF) gene by homologous recombination caused alveolar proteinosis in mice. To further…”
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16
Generation and characterization of urokinase receptor-deficient mice
Published in The Journal of clinical investigation (01-02-1996)“…Mice homozygously deficient for the urokinase-type plasminogen activator (u-PA) receptor (u-PAR-1-) were generated by homologous recombination in D3, embryonic…”
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17
Demonstration that CFTR is a Chloride Channel by Alteration of its Anion Selectivity
Published in Science (American Association for the Advancement of Science) (12-07-1991)“…Expression of the cystic fibrosis transmembrane conductance regulator (CFTR) generates adenosine 3′,5′-monophosphate (cAMP)-regulated chloride channels,…”
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18
Demonstration of a rational strategy for human prostate cancer gene therapy
Published in The Journal of urology (01-03-1994)“…The potential efficacy and clinical feasibility of gene therapy for prostate cancer were tested. Efficacy was tested using the Dunning rat prostate carcinoma…”
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19
Dispensable Sequence Motifs in the RAG-1 and RAG-2 Genes for Plasmid V(D)J Recombination
Published in Proceedings of the National Academy of Sciences - PNAS (01-07-1993)“…As a probe of whether RAG-1 and RAG-2 gene products activate other genes or form part of the recombinase itself, certain mutants of the RAG genes were assayed…”
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Generation of a High-Titer Retroviral Vector Capable of Expressing High Levels of the Human β-Globin Gene
Published in Proceedings of the National Academy of Sciences - PNAS (18-07-1995)“…Retrovirus-mediated gene transfer into hematopoietic cells may provide a means of treating both inherited and acquired diseases involving hematopoietic cells…”
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