Gene Therapy

Stem cell transplantation for primary immunodeficiencies

Quality of life / Gene Therapy / Stem Cell Transplantation / Italy / Humans / Natural Killer cells / Primary Immunodeficiency / Infant / Bone marrow / Registries / Clinical Sciences / Bone Marrow Transplantation / Success Rate / Time Factors / Survival Rate / Wiskott Aldrich Syndrome / Severe combined immunodeficiency / Hematopoietic Stem Cell Transplantation / Natural Killer cells / Primary Immunodeficiency / Infant / Bone marrow / Registries / Clinical Sciences / Bone Marrow Transplantation / Success Rate / Time Factors / Survival Rate / Wiskott Aldrich Syndrome / Severe combined immunodeficiency / Hematopoietic Stem Cell Transplantation

A combinatorial approach to create artificial homing endonucleases cleaving chosen sequences

Biology / Gene Therapy / Medicine / Protein Engineering / Biological Sciences / DNA / Environmental Sciences / Humans / Mutation / Nucleic Acids / Substrate Specificity / DNA binding / Gene Targeting / Genome sequence / Nucleotides / Dimerization / DNA / Environmental Sciences / Humans / Mutation / Nucleic Acids / Substrate Specificity / DNA binding / Gene Targeting / Genome sequence / Nucleotides / Dimerization

Intraarticular expression of biologically active interleukin 1-receptor-antagonist protein by ex vivo gene transfer

Gene Therapy / Gene expression / Multidisciplinary / Humans / Animals / Gene transfer techniques / Arthritis / Molecular cloning / Genetically Modified / Transfection / Biological activity / Rabbits / Knee Joint / Gene Transfer / Interleukin / Gene transfer techniques / Arthritis / Molecular cloning / Genetically Modified / Transfection / Biological activity / Rabbits / Knee Joint / Gene Transfer / Interleukin

Prolonged Biologically Active Transgene Expression Driven by HSV Latency Active Promoter 2 (LAP2) in Brain In Vivo

Pharmacology / Technology / Gene Therapy / Gene expression / Biological Sciences / Molecular / Brain / Low Dose / Mice / Chronic Disease / Female / Animals / Therapy / Substantia nigra / Rats / Biological activity / Transgene Expression / Molecular Targeted Therapy / Parkinson Disease / Herpes Simplex Virus / Molecular / Brain / Low Dose / Mice / Chronic Disease / Female / Animals / Therapy / Substantia nigra / Rats / Biological activity / Transgene Expression / Molecular Targeted Therapy / Parkinson Disease / Herpes Simplex Virus

Sequential delivery of interferon-α gene and DCs to intracranial gliomas promotes an effective antitumor response

Immune response / Gene Therapy / Biological Sciences / Dendritic cell / Lymph Node / Antitumor Activity

Increased expression of rapsyn in muscles prevents acetylcholine receptor loss in experimental autoimmune myasthenia gravis

Electron Microscopy / Skeletal muscle biology / Gene Therapy / Immunohistochemistry / Confocal Microscopy / Brain / Female / Animals / Disease Severity / Electromyography / Synaptic Transmission / Fluorescent Antibody Technique / Rats / Myasthenia Gravis / Human Disease / Action Potentials / Individual Difference / Neuromuscular Junction / Radioimmunoassay / Brain / Female / Animals / Disease Severity / Electromyography / Synaptic Transmission / Fluorescent Antibody Technique / Rats / Myasthenia Gravis / Human Disease / Action Potentials / Individual Difference / Neuromuscular Junction / Radioimmunoassay

Increased expression of rapsyn in muscles prevents acetylcholine receptor loss in experimental autoimmune myasthenia gravis

Electron Microscopy / Skeletal muscle biology / Gene Therapy / Immunohistochemistry / Confocal Microscopy / Brain / Female / Animals / Disease Severity / Electromyography / Synaptic Transmission / Fluorescent Antibody Technique / Rats / Myasthenia Gravis / Human Disease / Action Potentials / Individual Difference / Neuromuscular Junction / Radioimmunoassay / Brain / Female / Animals / Disease Severity / Electromyography / Synaptic Transmission / Fluorescent Antibody Technique / Rats / Myasthenia Gravis / Human Disease / Action Potentials / Individual Difference / Neuromuscular Junction / Radioimmunoassay

Correction of Laminin-5 Deficiency in Human Epidermal Stem Cells by Transcriptionally Targeted Lentiviral Vectors

Genetics / Technology / Stem Cells / Gene Therapy / Health / Stem Cell / Gene expression / Biological Sciences / Molecular / Cell Differentiation / Humans / Keratinocytes / Basement Membrane / Mice / Animals / Epidermolysis Bullosa / Lentivirus / Epidermis / Transgene Expression / Molecular Targeted Therapy / Gene Transfer / Cell Adhesion Molecules / Tissue Specificity / Skin Biopsy / Stem Cell / Gene expression / Biological Sciences / Molecular / Cell Differentiation / Humans / Keratinocytes / Basement Membrane / Mice / Animals / Epidermolysis Bullosa / Lentivirus / Epidermis / Transgene Expression / Molecular Targeted Therapy / Gene Transfer / Cell Adhesion Molecules / Tissue Specificity / Skin Biopsy

Cardiomyocyte Transplantation in a Porcine Myocardial Infarction Model

Technology / Electron Microscopy / Gene Therapy / Biological Sciences / Cell line / Animals / Cell Transplantation / Myocardial Infarction / Myocardium / Swine / Arrhythmia / Animals / Cell Transplantation / Myocardial Infarction / Myocardium / Swine / Arrhythmia

Mouse Model for Human Arginase Deficiency

Cellular Biology / Gene Therapy / Biological Sciences / Molecular and cellular biology / Humans / Kidney / Liver / Mice / Female / Animals / Male / Nitrogen / Detoxification / Enzyme / Human Genome / Mouse Model / Mammary gland / Enzyme activity / Homologous Recombination / Quaternary Ammonium Compounds / Knockout Mice / Glutamic Acid / Growth Retardation / Kidney / Liver / Mice / Female / Animals / Male / Nitrogen / Detoxification / Enzyme / Human Genome / Mouse Model / Mammary gland / Enzyme activity / Homologous Recombination / Quaternary Ammonium Compounds / Knockout Mice / Glutamic Acid / Growth Retardation

Gene transfer of human interferon gamma complementary DNA into a renal cell carcinoma line enhances MHC-restricted cytotoxic T lymphocyte recognition but suppresses non-MHC-restricted effector cell activity

Gene Therapy / Gene expression / Cancer Vaccines / Biological Sciences / Humans / Renal cell Carcinoma / Antigen Processing / Proteins / Gene / Multienzyme complexes / Cysteine endopeptidases / Transfection / MHC class I / Gene Transfer / Cell Adhesion Molecules / Interferon gamma / Renal cell Carcinoma / Antigen Processing / Proteins / Gene / Multienzyme complexes / Cysteine endopeptidases / Transfection / MHC class I / Gene Transfer / Cell Adhesion Molecules / Interferon gamma

Pyridinium cationic lipids in gene delivery: an in vitro and in vivo comparison of transfection efficiency versus a tetraalkylammonium congener

Gene Therapy / DNA / Lung Cancer / Lipids / Cell line / Humans / Animals / Gene transfer techniques / Liposomes / Drug Delivery Systems / Gene Delivery / In Vivo / Neoplasms / Gene Targeting / Transfection / Quaternary Ammonium Compounds / Cations / Nude mice / Biochemistry and cell biology / Gene Transfer / Humans / Animals / Gene transfer techniques / Liposomes / Drug Delivery Systems / Gene Delivery / In Vivo / Neoplasms / Gene Targeting / Transfection / Quaternary Ammonium Compounds / Cations / Nude mice / Biochemistry and cell biology / Gene Transfer

Transcription factor decoy for AP-1 reduces mesangial cell proliferation and extracellular matrix production in vitro and in vivo

Gene Therapy / Transcription Regulation / Gene expression / Extracellular Matrix / Cell Division / Biological Sciences / Glucose / Virus / Animals / Renal failure / Male / Transcription Factor / Diabetic Nephropathy / VECTOR / Gene Delivery / Rats / Transfection / Cell Proliferation / Streptozotocin / Base Sequence / Angiotensin II / Diabetic Rat / Plasminogen Activator Inhibitor / Gene Transfer / Molecular Sequence Data / Biological Sciences / Glucose / Virus / Animals / Renal failure / Male / Transcription Factor / Diabetic Nephropathy / VECTOR / Gene Delivery / Rats / Transfection / Cell Proliferation / Streptozotocin / Base Sequence / Angiotensin II / Diabetic Rat / Plasminogen Activator Inhibitor / Gene Transfer / Molecular Sequence Data

Hypoxia-induced human endonuclease G expression suppresses tumor growth in a xenograft model

Oncology / Biotechnology / Cancer / Fluorescence Microscopy / Gene Therapy / Prostate Cancer / Cell line / Humans / Anoxia / Mice / Female / Animals / Male / Cell Death / Cobalt / Cancer Gene Therapy / Time Dependent / Neoplasms / Growth rate / G protein / Cell Proliferation / Tumor Growth / Gene Transfer / Transient Expression / Prostate Cancer / Cell line / Humans / Anoxia / Mice / Female / Animals / Male / Cell Death / Cobalt / Cancer Gene Therapy / Time Dependent / Neoplasms / Growth rate / G protein / Cell Proliferation / Tumor Growth / Gene Transfer / Transient Expression

Enfermedades pulmonares intersticiales difusas

Gene Therapy / Interstitial Lung Disease / Clinical Sciences

High-level beta-globin expression after retroviral transfer of locus activation region-containing human beta-globin gene derivatives into murine erythroleukemia cells

Gene Therapy / Gene expression / Multidisciplinary / Transgenic Mice / Phosphorus / Cell line / Humans / Mice / Beta decay / Animals / Enzyme / Genes / Phosphodiesterase Inhibitors / Immune system / Transfection / Organic Compound / Gene Transfer / Nucleic Acid / Gene Expression Regulation / DNA sequence / Cell line / Humans / Mice / Beta decay / Animals / Enzyme / Genes / Phosphodiesterase Inhibitors / Immune system / Transfection / Organic Compound / Gene Transfer / Nucleic Acid / Gene Expression Regulation / DNA sequence
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