| Item Type | Name |
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Academic Article
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Sustained miRNA-mediated knockdown of mutant AAT with simultaneous augmentation of wild-type AAT has minimal effect on global liver miRNA profiles.
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Academic Article
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Gene transfer in the lung using recombinant adeno-associated virus.
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Academic Article
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Gene transfer in skeletal and cardiac muscle using recombinant adeno-associated virus.
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Concept
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Gene Expression Profiling
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Concept
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Enzyme-Linked Immunosorbent Assay
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Concept
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Extracellular Matrix
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Concept
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Neutrophils
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Concept
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Disease Models, Animal
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Concept
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Fibroblasts
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Concept
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Injections, Intramuscular
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Concept
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Blood Urea Nitrogen
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Concept
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Euthanasia, Animal
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Concept
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Humans
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Concept
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Animals, Newborn
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Concept
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Genetic Therapy
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Concept
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Actins
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Concept
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Gene Expression
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Concept
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Rare Diseases
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Concept
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Terminal Repeat Sequences
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Concept
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Lymphocyte Count
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Concept
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Amyotrophic Lateral Sclerosis
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Concept
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Dependovirus
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Concept
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Extremities
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Concept
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Immunophenotyping
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Concept
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Mice, Knockout
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Concept
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Seasons
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Concept
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Urination Disorders
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Concept
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Pyelonephritis
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Concept
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Blotting, Western
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Concept
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T-Lymphocytes, Regulatory
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Concept
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Arteries
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Concept
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Anti-Inflammatory Agents, Non-Steroidal
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Concept
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T-Lymphocyte Subsets
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Concept
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Biopsy
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Concept
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Clonixin
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Concept
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Mice
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Concept
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Founder Effect
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Concept
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Clinical Trials as Topic
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Concept
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Aging
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Concept
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Endopeptidases
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Concept
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Lung Diseases
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Concept
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Albendazole
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Concept
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Cattle
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Concept
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Transgenes
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Concept
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Mice, Inbred C57BL
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Concept
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Gene Expression Regulation
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Concept
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DNA Repeat Expansion
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Concept
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Mutation
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Concept
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Cells, Cultured
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Concept
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Veins
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Concept
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Mice, Transgenic
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Concept
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Containment of Biohazards
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Concept
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Capsid
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Concept
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Cell Proliferation
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Concept
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alpha 1-Antitrypsin
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Concept
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Primates
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Concept
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Air Pollutants, Occupational
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Concept
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Macaca mulatta
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Concept
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Severe Combined Immunodeficiency
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Concept
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Retina
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Concept
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Green Fluorescent Proteins
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Concept
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Environmental Monitoring
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Concept
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Horses
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Concept
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Gene Order
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Concept
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Stress, Mechanical
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Concept
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Pancreatic Elastase
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Concept
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Orbit
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Concept
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Maple Syrup Urine Disease
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Concept
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Camelids, New World
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Concept
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Dipeptides
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Concept
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Transduction, Genetic
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Concept
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Muscular Atrophy, Spinal
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Concept
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Rats
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Concept
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Housing, Animal
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Concept
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Collagen
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Concept
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alpha 1-Antitrypsin Deficiency
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Concept
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Oligonucleotide Array Sequence Analysis
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Concept
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Genetic Vectors
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Concept
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Anthelmintics
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Concept
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MicroRNAs
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Concept
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Hydrogen-Ion Concentration
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Concept
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Promoter Regions, Genetic
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Concept
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INDEL Mutation
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Concept
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Gene Silencing
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Concept
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Base Sequence
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Concept
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Alleles
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Concept
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Gene Knockdown Techniques
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Concept
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3-Methyl-2-Oxobutanoate Dehydrogenase (Lipoamide)
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Concept
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Immunohistochemistry
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Concept
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Injections, Intravenous
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Concept
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Gene Transfer Techniques
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Concept
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Pulmonary Emphysema
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Concept
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Aerosols
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Academic Article
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Stability and compatibility of recombinant adeno-associated virus under conditions commonly encountered in human gene therapy trials.
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Academic Article
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Progress with Recombinant Adeno-Associated Virus Vectors for Gene Therapy of Alpha-1 Antitrypsin Deficiency.
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Academic Article
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Delivery of Adeno-Associated Virus Gene Therapy by Intravascular Limb Infusion Methods.
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Academic Article
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A comparison of total, respirable, and real-time airborne particulate sampling in horse barns.
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Academic Article
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Urinary bladder agenesis in an alpaca (Vicugna pacos) cria.
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Academic Article
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Age-dependent decline in mouse lung regeneration with loss of lung fibroblast clonogenicity and increased myofibroblastic differentiation.
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Academic Article
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Age dependence of lung mesenchymal stromal cell dynamics following pneumonectomy.
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Academic Article
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Presumptive albendazole toxicosis in 12 alpacas.
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Academic Article
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Retro-Orbital Venous Sinus Delivery of rAAV9 Mediates High-Level Transduction of Brain and Retina Compared with Temporal Vein Delivery in Neonatal Mouse Pups.
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Academic Article
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Cellular kinetics and modeling of bronchioalveolar stem cell response during lung regeneration.
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Academic Article
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Matrix modulation of compensatory lung regrowth and progenitor cell proliferation in mice.
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Academic Article
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5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency.
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Academic Article
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Editing out five Serpina1 paralogs to create a mouse model of genetic emphysema.
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Academic Article
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Quantification of Total Human Alpha-1 Antitrypsin by Sandwich ELISA.
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Academic Article
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Genotyping Protocol for the Alpha-1 Antitrypsin (PiZ) Mouse Model.
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Academic Article
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Therapeutics: Gene Therapy for Alpha-1 Antitrypsin Deficiency.
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Academic Article
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Bridging from Intramuscular to Limb Perfusion Delivery of rAAV: Optimization in a Non-human Primate Study.
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Academic Article
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The rapidly evolving state of gene therapy.
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Academic Article
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Muscle-Directed Delivery of an AAV1 Vector Leads to Capsid-Specific T Cell Exhaustion in Nonhuman Primates and Humans.
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Academic Article
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Therapeutic plasma exchange to mitigate flunixin meglumine overdose in a cria.
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Academic Article
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Modulating immune responses to AAV by expanded polyclonal T-regs and capsid specific chimeric antigen receptor T-regulatory cells.
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Concept
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Gene Editing
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|
Concept
|
Transcriptome
|
|
Concept
|
Myofibroblasts
|
|
Concept
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Epidemiological Monitoring
|
|
Concept
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Genotyping Techniques
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|
Concept
|
Drug Overdose
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Academic Article
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Gene Therapy and the Use of Animal Models: Why Mice Alone Are Not Sufficient.
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Academic Article
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Intratracheally administered LNA gapmer antisense oligonucleotides induce robust gene silencing in mouse lung fibroblasts.
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Academic Article
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Gene therapy for alpha-1 antitrypsin deficiency: an update.
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Academic Article
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Artificial microRNA suppresses C9ORF72 variants and decreases toxic dipeptide repeat proteins in vivo.
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Academic Article
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Alpha-1 Antitrypsin Deficiency.
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Academic Article
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Approaches to Therapeutic Gene Editing in Alpha-1 Antitrypsin Deficiency.
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Academic Article
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Serum Western Blot for the Detection of a c-Myc Protein Tag in Non-human Primates and Mice.
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Academic Article
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Improved gene therapy for spinal muscular atrophy in mice using codon-optimized hSMN1 transgene and hSMN1 gene-derived promotor.
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Academic Article
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Biodistribution and safety of a single rAAV3B-AAT vector for silencing and replacement of alpha-1 antitrypsin in Cynomolgus macaques.
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Academic Article
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Limb Perfusion Delivery of a rAAV1 Alpha-1 Antitrypsin Vector in Non-Human Primates Is Safe but Insufficient for Therapy.
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Academic Article
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BCKDHA-BCKDHB digenic gene therapy restores metabolic homeostasis in two mouse models and a calf with classic maple syrup urine disease.
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