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1. Full Text In vivo genome editing of the albumin locus as a platform for protein replacement therapy
by Sharma, Rajiv and Anguela, Xavier M and Doyon, Yannick and Wechsler, Thomas and DeKelver, Russell C and Sproul, Scott and Paschon, David E and Miller, Jeffrey C and Davidson, Robert J and Shivak, David and Zhou, Shangzhen and Rieders, Julianne and Gregory, Philip D and Holmes, Michael C and Rebar, Edward J and High, Katherine A
Blood, ISSN 0006-4971, 10/2015, Volume 126, Issue 15, pp. 1777 - 1784
Life Sciences & Biomedicine | Hematology | Science & Technology | Hemophilia B - therapy | Dependovirus - genetics | Genetic Therapy | Transgenes - physiology | Genetic Vectors - administration & dosage | Fabry Disease - genetics | RNA Editing | Humans | Lysosomes - enzymology | Gaucher Disease - therapy | Promoter Regions, Genetic - genetics | Mucopolysaccharidosis I - therapy | Mucopolysaccharidosis II - therapy | Mucopolysaccharidosis II - genetics | Real-Time Polymerase Chain Reaction | Enzyme Replacement Therapy | Zinc Fingers | Albumins - metabolism | Liver - metabolism | Mice, Inbred C57BL | RNA, Messenger - genetics | Factor VIII - genetics | Factor IX - genetics | Hemophilia B - genetics | Hemophilia A - therapy | Reverse Transcriptase Polymerase Chain Reaction | Animals | Gaucher Disease - genetics | Endonucleases | Mucopolysaccharidosis I - genetics | Hemophilia A - genetics | High-Throughput Nucleotide Sequencing | Mice | Fabry Disease - therapy | Genome | Albumins - genetics | Index Medicus | Abridged Index Medicus | Gene Therapy
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2. Full Text In vivo genome editing restores haemostasis in a mouse model of haemophilia
by Li, Hojun and Haurigot, Virginia and Doyon, Yannick and Li, Tianjian and Wong, Sunnie Y and Bhagwat, Anand S and Malani, Nirav and Anguela, Xavier M and Sharma, Rajiv and Ivanciu, Lacramiora and Murphy, Samuel L and Finn, Jonathan D and Khazi, Fayaz R and Zhou, Shangzhen and Paschon, David E and Rebar, Edward J and Bushman, Frederic D and Gregory, Philip D and Holmes, Michael C and High, Katherine A
Nature (London), ISSN 0028-0836, 07/2011, Volume 475, Issue 7355, pp. 217 - 221
Science & Technology - Other Topics | Multidisciplinary Sciences | Science & Technology | Hematologic and hematopoietic diseases | Biological and medical sciences | Medical sciences | Platelet diseases and coagulopathies | Gene Targeting - methods | Endonucleases - genetics | Factor IX - analysis | Humans | Genome - genetics | DNA Repair - genetics | Endonucleases - metabolism | DNA Breaks, Double-Stranded | Sequence Homology | Hemostasis | Base Sequence | HEK293 Cells | Disease Models, Animal | Liver Regeneration | Zinc Fingers | Introns - genetics | Liver - metabolism | Mice, Inbred C57BL | Exons - genetics | Factor IX - genetics | Hemophilia B - genetics | Mutation - genetics | Genetic Vectors - genetics | Endonucleases - chemistry | Phenotype | Animals | Hemophilia B - physiopathology | Cell Line, Tumor | Mice | Genetic Therapy - methods | Hemophilia | Physiological aspects | Genomes | Genetic aspects | Research | Health aspects | Genetic disorders | Rodents | Stem cells | Editing | Genetic engineering | Mutation | Gene therapy | Gene expression | Index Medicus
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3. Full Text Engineering adeno-associated viruses for clinical gene therapy
by Kotterman, Melissa A and Schaffer, David V
Nature reviews. Genetics, ISSN 1471-0056, 2014, Volume 15, Issue 7, pp. 445 - 451
Life Sciences & Biomedicine | Genetics & Heredity | Science & Technology | Dependovirus - genetics | Genome, Viral | Metabolic Diseases - pathology | Capsid - chemistry | Muscular Dystrophies - therapy | Humans | Viral Proteins - genetics | Clinical Trials as Topic | Directed Molecular Evolution | Hemophilia A - therapy | Muscular Dystrophies - genetics | Muscular Dystrophies - pathology | Hemophilia A - pathology | Metabolic Diseases - therapy | Genetic Engineering | Metabolic Diseases - genetics | Hemophilia A - genetics | Capsid - metabolism | DNA, Viral - genetics | Genetic Vectors | Genetic Therapy - methods | Usage | Microbial genetic engineering | Genetic aspects | Dependoviruses | Gene therapy | Health aspects | Methods | Index Medicus
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4. Full Text A Novel Platform for Immune Tolerance Induction in Hemophilia A Mice
by Merlin, Simone and Cannizzo, Elvira Stefania and Borroni, Ester and Bruscaggin, Valentina and Schinco, Piercarla and Tulalamba, Warut and Chuah, Marinee K and Arruda, Valder R and VandenDriessche, Thierry and Prat, Maria and Valente, Guido and Follenzi, Antonia
Molecular therapy, ISSN 1525-0016, 08/2017, Volume 25, Issue 8, pp. 1815 - 1830
hemophilia A | gene therapy | targeted FVIII expression | inhibitor titers reversion | Tregs | Genetics & Heredity | Life Sciences & Biomedicine | Biotechnology & Applied Microbiology | Medicine, Research & Experimental | Science & Technology | Research & Experimental Medicine | Factor VIII - immunology | T-Lymphocytes, Regulatory - metabolism | Immunosuppression - methods | Male | Mice, 129 Strain | T-Lymphocytes, Regulatory - immunology | Isoantibodies - blood | Organ Specificity - genetics | CD11b Antigen - genetics | Lentivirus - genetics | Transgenes | Genes, Reporter | Disease Models, Animal | Promoter Regions, Genetic | Gene Expression | Immunization | Transduction, Genetic | Endothelial Cells - metabolism | Hemophilia A - immunology | Mice, Inbred C57BL | Factor VIII - genetics | Genetic Vectors - genetics | Animals | Immune Tolerance - genetics | Isoantibodies - immunology | Hemophilia A - genetics | Mice | Mice, Inbred BALB C | Factor VIII - metabolism | Whole Blood Coagulation Time | Myeloid cells | Immunoglobulins | Liver | Cloning | Lymphocytes T | Experiments | Immunological tolerance | Endothelial cells | Hemopoiesis | Coagulation factors | Hepatocytes | MicroRNAs | Hemophilia | Conflicts of interest | miRNA | Software | Factor VIII deficiency | Gene therapy | Index Medicus | Original
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5. 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
Life Sciences & Biomedicine | Genetics & Heredity | Science & Technology | 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 | Index Medicus
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6. Full Text Gene Therapy: Safer and Virus-Free?
by Dan Ferber
Science (American Association for the Advancement of Science), ISSN 0036-8075, 11/2001, Volume 294, Issue 5547, pp. 1638 - 1642
Genetic vectors | Plasmids | DNA | Genes | Liver | Genetic research | Clinical trials | Viruses | News Focus | Gene therapy | Liver cells | Science & Technology - Other Topics | Multidisciplinary Sciences | Science & Technology | Blood Pressure | Viruses - pathogenicity | Viruses - genetics | Genetic Vectors - administration & dosage | Muscular Dystrophies - therapy | Humans | Genetic Vectors - adverse effects | Muscular Dystrophies - genetics | RNA - genetics | Transfection | Time Factors | Melanoma - genetics | Plasmids - genetics | Gene Expression | Microinjections | Transgenes - genetics | Biolistics | DNA - administration & dosage | Gene Transfer Techniques - trends | Electroporation | Clinical Trials as Topic | Genetic Therapy - trends | Hemophilia A - therapy | Organ Specificity | Genetic Vectors - genetics | DNA - genetics | Animals | Coronary Artery Disease - therapy | Coronary Artery Disease - genetics | Hemophilia A - genetics | RNA - administration & dosage | Melanoma - therapy | Plasmids - administration & dosage | Genetic Therapy - methods | Injections, Intra-Arterial | Proteins | Deoxyribonucleic acid
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7. Full Text Advances and challenges for hemophilia gene therapy
by Batty, Paul and Lillicrap, David
Human molecular genetics, ISSN 0964-6906, 10/2019, Volume 28, Issue R1, pp. R95 - R101
Biochemistry & Molecular Biology | Genetics & Heredity | Life Sciences & Biomedicine | Science & Technology | Hemophilia B - therapy | Transduction, Genetic | Humans | Gene Expression Regulation | Factor VIII - genetics | Factor IX - genetics | Genetic Vectors - adverse effects | Treatment Outcome | Combined Modality Therapy | Hemophilia B - genetics | Hemophilia A - therapy | Organ Specificity | Genetic Vectors - genetics | Clinical Studies as Topic | Animals | Isoantibodies - immunology | Hemophilia A - genetics | Transgenes | Genetic Therapy - adverse effects | Genetic Therapy - methods | Index Medicus
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