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1. Full Text Ex vivo gene transfer and correction for cell-based therapies
by Naldini, Luigi
Nature Reviews Genetics, ISSN 1471-0056, 05/2011, Volume 12, Issue 5, pp. 301 - 315
Cell-based therapies are fast-growing forms of personalized medicine that make use of the steady advances in stem cell manipulation and gene transfer... 
CHRONIC GRANULOMATOUS-DISEASE | BONE-MARROW-TRANSPLANTATION | HEMATOPOIETIC STEM-CELLS | IN-VIVO | GENETICS & HEREDITY | CENTRAL-NERVOUS-SYSTEM | NOD/SCID REPOPULATING CELLS | ZINC-FINGER NUCLEASES | LENTIVIRAL VECTOR INTEGRATION | VERSUS-HOST-DISEASE | CD8(+) T-CELLS | Gene Transfer Techniques | Animals | Humans | Dogs | Clinical Trials as Topic | Mutagenesis, Insertional | Hematopoietic Stem Cell Transplantation - methods | Genetic Therapy - methods | Usage | Stem cells | Genetic engineering | Transplantation | Gene therapy | Health aspects | Methods
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2. Full Text Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges
by High, Katherine A and Mingozzi, Federico
Nature Reviews Genetics, ISSN 1471-0056, 05/2011, Volume 12, Issue 5, pp. 341 - 355
In vivo gene replacement for the treatment of inherited disease is one of the most compelling concepts in modern medicine. Adeno-associated virus (AAV) vectors... 
ADENOASSOCIATED VIRUS TYPE-2 | SKELETAL-MUSCLE | HUMAN-FACTOR-IX | LEBER CONGENITAL AMAUROSIS | LONG-TERM CORRECTION | LYSOSOMAL STORAGE DISEASE | SUSTAINED PHENOTYPIC CORRECTION | CANINE HEMOPHILIA-B | GENETICS & HEREDITY | PHASE-I | DEFICIENT MOUSE MODEL | Hemophilia B - therapy | Dependovirus - genetics | Gene Transfer Techniques | Retinal Diseases - genetics | Humans | Retinal Diseases - therapy | Rats | Male | Clinical Trials as Topic | Genetic Diseases, Inborn - therapy | Hemophilia B - genetics | Animals | Dogs | Mice | Genetic Vectors | Genetic Therapy - methods | Care and treatment | Usage | Genetic transformation | Hemophilia | Genetic aspects | Research | Dependoviruses | Gene therapy | Health aspects | Risk factors | Methods
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3. Full Text Ex vivo gene transfer for improved adoptive immunotherapy of cancer
by Ngo, Minhtran C and Rooney, Cliona M and Howard, Jeffrey M and Heslop, Helen E
Human Molecular Genetics, ISSN 0964-6906, 04/2011, Volume 20, Issue R1, pp. R93 - R99
Adoptive immunotherapy is an appealing approach to cancer treatment, with the potential for more precise targeting and reduced toxicity. While early clinical... 
NATURAL-KILLER-CELLS | NK CELLS | CLINICAL-TRIAL | BIOCHEMISTRY & MOLECULAR BIOLOGY | GENETICS & HEREDITY | CHIMERIC ANTIGEN RECEPTOR | STEM-CELL TRANSPLANTATION | ANTITUMOR-ACTIVITY | VERSUS-HOST-DISEASE | SUICIDE-GENE | PHASE-I | CYTOTOXIC T-LYMPHOCYTES | Melanoma - metabolism | Gene Transfer Techniques | Immunotherapy, Adoptive - methods | Receptors, Antigen, T-Cell - metabolism | Humans | Neoplasms - therapy | Animals | Neoplasms - genetics | T-Lymphocytes - metabolism | Melanoma - immunology | Neoplasms - immunology | T-Lymphocytes - immunology | Melanoma - therapy | Reviews
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4. Full Text Strategies to Improve DNA Polyplexes for in Vivo Gene Transfer: Will "Artificial Viruses" Be the Answer?
by Wagner, Ernst
Pharmaceutical Research, ISSN 0724-8741, 01/2004, Volume 21, Issue 1, pp. 8 - 14
For the purpose of introducing nucleic acids into cells, cationic polymers have been steadily improved as gene carriers. This has resulted in improved... 
Nonviral vectors | Gene delivery | Cationic polymers | Targeted gene therapy | cationic polymers | DELIVERY-SYSTEMS | COMPLEXES | LOW-MOLECULAR-WEIGHT | LINEAR POLYETHYLENIMINE | RECEPTOR | CHEMISTRY, MULTIDISCIPLINARY | gene delivery | targeted gene therapy | INVIVO | PLASMID DNA | POLYMER | PHARMACOLOGY & PHARMACY | nonviral vectors | EXPRESSION | SYSTEMIC CIRCULATION | DNA - genetics | Polymers - administration & dosage | Animals | Genetic Vectors - administration & dosage | Humans | DNA - administration & dosage | Polymers - adverse effects | Gene Transfer Techniques - adverse effects | DNA Viruses - genetics | Genetic Therapy - adverse effects | Genetic Therapy - methods
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5. Full Text Bone Regeneration by Regulated In Vivo Gene Transfer Using Biocompatible Polyplex Nanomicelles
by Itaka, Keiji and Ohba, Shinsuke and Miyata, Kanjiro and Kawaguchi, Hiroshi and Nakamura, Kozo and Takato, Tsuyoshi and Chung, Ung-Il and Kataoka, Kazunori
Molecular Therapy, ISSN 1525-0016, 09/2007, Volume 15, Issue 9, pp. 1655 - 1662
Gene therapy is a promising strategy for bone regenerative medicine. Although viral vectors have been intensively studied for delivery of osteogenic factors,... 
MEDICINE, RESEARCH & EXPERIMENTAL | CELLS | FIBROBLASTS | DELIVERY | POLYION COMPLEX MICELLES | THERAPY | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | PLASMID DNA | GENETICS & HEREDITY | BMP-2 GENE | DIFFERENTIATION | MORPHOGENETIC PROTEIN-2 | NONVIRAL VECTORS | Bone Regeneration - physiology | Cell Line | Gene Transfer Techniques | Genetic Vectors - administration & dosage | Genetic Vectors - chemistry | Bone Regeneration - genetics | Biocompatible Materials - chemistry | Humans | Core Binding Factor Alpha 1 Subunit - physiology | Bone Morphogenetic Protein Receptors, Type I - genetics | Polyethylene Glycols - chemistry | Genetic Vectors - genetics | Bone Morphogenetic Protein Receptors, Type I - physiology | Animals | Micelles | Polyethyleneimine - chemistry | Mice | Core Binding Factor Alpha 1 Subunit - genetics | Nanotechnology | Genetic Therapy - methods
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6. Full Text Performance of High Quality Minicircle DNA for In Vitro and In Vivo Gene Transfer
by Kobelt, Dennis and Kobelt, Dennis and Schleef, Martin and Schleef, Martin and Schmeer, Marco and Schmeer, Marco and Aumann, Jutta and Aumann, Jutta and Schlag, Peter M and Schlag, Peter M and Walther, Wolfgang and Walther, Wolfgang
Molecular Biotechnology, ISSN 1073-6085, 1/2013, Volume 53, Issue 1, pp. 80 - 89
Plasmid DNA is frequently used particularly for nonviral gene therapy. Conventional plasmid DNA contains bacterial backbone and resistance gene sequences, as... 
Biochemistry, general | Protein Science | Human Genetics | Biotechnology | Chemistry | Nonviral gene transfer | Biological Techniques | Minicircle DNA | Plasmid DNA | Gene therapy | Cell Biology | Cancer | SYSTEM | PLASMIDS | PERSISTENT | SEQUENCES | BIOCHEMISTRY & MOLECULAR BIOLOGY | DELIVERY | SITE-SPECIFIC RECOMBINATION | JET-INJECTION TECHNOLOGY | THERAPY | EPISOMAL TRANSGENE EXPRESSION | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | VECTORS | Gene Transfer Techniques | Genetic Therapy | Gene Expression | Luciferases - metabolism | Humans | DNA, Circular - genetics | Electroporation | Gene Dosage | Sequence Analysis, DNA | Luciferases - genetics | Animals | Electrophoresis, Agar Gel | Plasmids | Recombination, Genetic | Cell Line, Tumor | Mice | Genetic Vectors | Transgenes | Real-Time Polymerase Chain Reaction | Colon cancer | Genetic vectors | RNA | Analysis | Genes | DNA | Luciferase | Melanoma | Genetic research | Gene expression | Deoxyribonucleic acid--DNA
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7. Full Text Robust in vivo Gene Transfer into Adult Mammalian Neural Stem Cells by Lentiviral Vectors
by Antonella Consiglio and Angela Gritti and Diego Dolcetta and Antonia Follenzi and Claudio Bordignon and Fred H. Gage and Angelo Luigi Vescovi and Luigi Naldini and Inder M. Verma
Proceedings of the National Academy of Sciences of the United States of America, ISSN 0027-8424, 10/2004, Volume 101, Issue 41, pp. 14835 - 14840
Stable genetic modification of adult stem cells is fundamental for both developmental studies and therapeutic purposes. Using in vivo marking studies, we... 
Biological Sciences | Neuroscience | Genetic vectors | Neurons | B lymphocytes | Multipotent stem cells | Cell lines | Stem cells | Neural stem cells | Cultured cells | Cells | PROGENITOR CELLS | SUBEPENDYMAL CELLS | OLFACTORY-BULB | MOUSE BRAIN | MULTIDISCIPLINARY SCIENCES | NEURONAL PRECURSORS | CENTRAL-NERVOUS-SYSTEM | SUBVENTRICULAR ZONE | GROWTH-FACTOR | DENTATE GYRUS | T-LYMPHOCYTES | Immunohistochemistry | Gene Transfer Techniques | Brain - cytology | Recombinant Proteins - analysis | Neurons - cytology | Stem Cells - cytology | Brain - physiology | Animals | Transfection - methods | Fluorescent Antibody Technique | Neurons - physiology | Lentivirus - genetics | Stem Cells - physiology | Mice | Cell Culture Techniques | Genetic Vectors | Genes, Reporter | Research
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