Item Type | Name |
Academic Article
|
Adenovirus-adeno-associated virus hybrid for large-scale recombinant adeno-associated virus production.
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Academic Article
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rAAV9--a human-derived adeno-associated virus vector for efficient transgene expression in mouse cingulate cortex.
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Academic Article
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MicroRNA-regulated, systemically delivered rAAV9: a step closer to CNS-restricted transgene expression.
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Academic Article
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Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system.
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Academic Article
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Exploiting natural diversity of AAV for the design of vectors with novel properties.
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Academic Article
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Long-term, efficient inhibition of microRNA function in mice using rAAV vectors.
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Academic Article
|
Gene transfer in the lung using recombinant adeno-associated virus.
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Academic Article
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SU9518 inhibits proliferative vitreoretinopathy in fibroblast and genetically modified M?ller cell-induced rabbit models.
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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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Academic Article
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Recombinant adeno-associated virus integration sites in murine liver after ornithine transcarbamylase gene correction.
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Academic Article
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Vector sequences are not detected in tumor tissue from research subjects with ornithine transcarbamylase deficiency who previously received adenovirus gene transfer.
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Academic Article
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Transendocardial delivery of AAV6 results in highly efficient and global cardiac gene transfer in rhesus macaques.
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Academic Article
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Production and discovery of novel recombinant adeno-associated viral vectors.
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Academic Article
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Retinal gene delivery by rAAV and DNA electroporation.
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Academic Article
|
Gene therapy for Canavan's disease takes a step forward.
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Academic Article
|
Gene transfer to the CNS using recombinant adeno-associated virus.
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Academic Article
|
Gene transfer in the liver using recombinant adeno-associated virus.
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Concept
|
Angiogenesis Inhibitors
|
Concept
|
Vascular Endothelial Growth Factor A
|
Concept
|
Ornithine Carbamoyltransferase
|
Concept
|
Disease Models, Animal
|
Concept
|
Fibroblasts
|
Concept
|
Injections, Intramuscular
|
Concept
|
Targeted Gene Repair
|
Concept
|
Retinal Pigment Epithelium
|
Concept
|
Inverted Repeat Sequences
|
Concept
|
Virus Cultivation
|
Concept
|
Sequence Analysis, DNA
|
Concept
|
Spastic Paraplegia, Hereditary
|
Concept
|
PPAR alpha
|
Concept
|
Brain Neoplasms
|
Concept
|
Osteoclasts
|
Concept
|
Interferon-beta
|
Concept
|
Prefrontal Cortex
|
Concept
|
Bone Resorption
|
Concept
|
Blood-Brain Barrier
|
Concept
|
Myelin Sheath
|
Concept
|
Cell Lineage
|
Concept
|
Nicotine
|
Concept
|
Humans
|
Concept
|
Animals, Newborn
|
Concept
|
Mitochondria
|
Concept
|
Prostatic Neoplasms
|
Concept
|
HeLa Cells
|
Concept
|
Heterozygote
|
Concept
|
Genetic Therapy
|
Concept
|
Osteocytes
|
Concept
|
Cilia
|
Concept
|
Cell Division
|
Concept
|
Gene Expression
|
Concept
|
Genes, MHC Class II
|
Concept
|
Papillomavirus Infections
|
Concept
|
Mutagenesis, Site-Directed
|
Concept
|
Terminal Repeat Sequences
|
Concept
|
Cats
|
Concept
|
RNA Interference
|
Concept
|
Colposcopy
|
Concept
|
Histone Deacetylase 1
|
Concept
|
Gyrus Cinguli
|
Concept
|
Mice, Inbred BALB C
|
Concept
|
Alveolar Bone Loss
|
Concept
|
Habenula
|
Concept
|
RNA Editing
|
Concept
|
Mice, Obese
|
Concept
|
Influenza A Virus, H1N1 Subtype
|
Concept
|
Wool
|
Concept
|
Amyotrophic Lateral Sclerosis
|
Concept
|
Hereditary Sensory and Autonomic Neuropathies
|
Concept
|
Ribonuclease III
|
Concept
|
Leber Congenital Amaurosis
|
Concept
|
Down-Regulation
|
Concept
|
Electroporation
|
Concept
|
Injections
|
Concept
|
Dependovirus
|
Concept
|
Treatment Outcome
|
Concept
|
Acetyl-CoA Carboxylase
|
Concept
|
Endonucleases
|
Concept
|
Osteoblasts
|
Concept
|
Amidohydrolases
|
Concept
|
China
|
Concept
|
X-Ray Diffraction
|
Concept
|
Canavan Disease
|
Concept
|
Palmitates
|
Concept
|
Sterol Regulatory Element Binding Protein 1
|
Concept
|
Encephalomyelitis, Autoimmune, Experimental
|
Concept
|
Osteogenesis
|
Concept
|
Adenosine Triphosphate
|
Concept
|
Drug Delivery Systems
|
Concept
|
Random Allocation
|
Concept
|
Mice, Knockout
|
Concept
|
Baculoviridae
|
Concept
|
Oxidative Phosphorylation
|
Concept
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Acetyl Coenzyme A
|
Concept
|
Antiviral Agents
|
Concept
|
Mesencephalon
|
Concept
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Colorectal Neoplasms
|
Concept
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CHO Cells
|
Concept
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Infusion Pumps, Implantable
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Concept
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Heart Failure
|
Concept
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Cricetulus
|
Concept
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Pneumonia, Viral
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Concept
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AMP-Activated Protein Kinases
|
Concept
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Fallopian Tubes
|
Concept
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Infusions, Intravenous
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Concept
|
Ornithine Carbamoyltransferase Deficiency Disease
|
Concept
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Up-Regulation
|
Concept
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Obesity
|
Concept
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Energy Metabolism
|
Concept
|
Monomeric GTP-Binding Proteins
|
Concept
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Mice
|
Concept
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Clinical Trials as Topic
|
Concept
|
Mucopolysaccharidosis VII
|
Concept
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Insulin Resistance
|
Concept
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Epilepsy
|
Concept
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Thalamus
|
Concept
|
Blastocyst
|
Concept
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Electrons
|
Concept
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Adiponectin
|
Concept
|
Gene Expression Regulation, Developmental
|
Concept
|
ATP Citrate (pro-S)-Lyase
|
Concept
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Cattle
|
Concept
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Transgenes
|
Concept
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Diabetes Mellitus, Type 2
|
Concept
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Mice, Inbred C57BL
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Concept
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Congenital Disorders of Glycosylation
|
Concept
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Haloperidol
|
Concept
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Gene Expression Regulation
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Concept
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Computational Biology
|
Concept
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Sequence Deletion
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Concept
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Prostate
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Concept
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Caudate Nucleus
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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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Liver Neoplasms
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Concept
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Mice, Transgenic
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Concept
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Uterine Cervical Neoplasms
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Concept
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Ubiquinone
|
Concept
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Huntington Disease
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Concept
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Containment of Biohazards
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Concept
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Oxygen
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Concept
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Capsid
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Concept
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Sheep, Domestic
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Concept
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Activins
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Concept
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Injections, Intraocular
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Concept
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Rod Cell Outer Segment
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Concept
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Nucleic Acid Conformation
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Concept
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Xenograft Model Antitumor Assays
|
Concept
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beta Catenin
|
Concept
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alpha 1-Antitrypsin
|
Concept
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Vaccines
|
Concept
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Primates
|
Concept
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DNA, Viral
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Concept
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Genetic Diseases, Inborn
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Concept
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Early Detection of Cancer
|
Concept
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Rabbits
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Concept
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Sirtuin 1
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Concept
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Codon, Terminator
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Concept
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Adaptor Proteins, Signal Transducing
|
Concept
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Receptors, Complement 3b
|
Concept
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Genetic Engineering
|
Concept
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Electrophoresis, Agar Gel
|
Concept
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Macaca mulatta
|
Concept
|
Tyrosinemias
|
Concept
|
DEAD-box RNA Helicases
|
Concept
|
Vero Cells
|
Concept
|
Genes, Reporter
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Concept
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Synapses
|
Concept
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Uterine Cervical Dysplasia
|
Concept
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Cerebrospinal Fluid
|
Concept
|
Cryoelectron Microscopy
|
Concept
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Corticotropin-Releasing Hormone
|
Concept
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Retina
|
Concept
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Epigenesis, Genetic
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Concept
|
Survival Analysis
|
Concept
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Arthritis, Rheumatoid
|
Concept
|
ATP-Binding Cassette Transporters
|
Concept
|
Electrolytes
|
Concept
|
Green Fluorescent Proteins
|
Concept
|
Gene Order
|
Concept
|
Recombinant Fusion Proteins
|
Concept
|
Osteogenesis Imperfecta
|
Concept
|
Interleukin-1beta
|
Concept
|
Sheep
|
Concept
|
Cocaine
|
Concept
|
Sequence Alignment
|
Concept
|
Fibroblast Growth Factors
|
Concept
|
Influenza Vaccines
|
Concept
|
Maple Syrup Urine Disease
|
Concept
|
Transduction, Genetic
|
Concept
|
Carcinoma, Hepatocellular
|
Concept
|
Receptors, Corticotropin-Releasing Hormone
|
Concept
|
Papillomaviridae
|
Concept
|
Internal Capsule
|
Concept
|
Mice, Nude
|
Concept
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Adenoviridae
|
Concept
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Case-Control Studies
|
Concept
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Muscular Atrophy, Spinal
|
Concept
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Histocompatibility Antigens Class II
|
Concept
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Gene Expression Regulation, Neoplastic
|
Concept
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Reproducibility of Results
|
Concept
|
Fumarates
|
Concept
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Clozapine
|
Concept
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Foam Cells
|
Concept
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Peptide-N4-(N-acetyl-beta-glucosaminyl) Asparagine Amidase
|
Concept
|
Mucopolysaccharidosis II
|
Concept
|
Electron Transport
|
Concept
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Stem Cell Transplantation
|
Concept
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Malonyl Coenzyme A
|
Concept
|
Antigen-Presenting Cells
|
Concept
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Cross-Sectional Studies
|
Concept
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Rats
|
Concept
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PPAR gamma
|
Concept
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Succinate Dehydrogenase
|
Concept
|
alpha 1-Antitrypsin Deficiency
|
Concept
|
Vitreous Body
|
Concept
|
Codon, Nonsense
|
Concept
|
Neostriatum
|
Concept
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Retinal Cone Photoreceptor Cells
|
Concept
|
DNA Replication
|
Concept
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Multiple Sclerosis
|
Concept
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RNA Splicing
|
Concept
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Callithrix
|
Concept
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Cell Line
|
Concept
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Transfection
|
Concept
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Coronavirus Infections
|
Concept
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Polymerase Chain Reaction
|
Concept
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Severe Acute Respiratory Syndrome
|
Concept
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Host-Pathogen Interactions
|
Concept
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Corpus Striatum
|
Concept
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Serotyping
|
Concept
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Gene Deletion
|
Concept
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Cholesterol
|
Concept
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Complement C3
|
Concept
|
Genetic Vectors
|
Concept
|
Putamen
|
Concept
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Ventral Tegmental Area
|
Concept
|
MicroRNAs
|
Concept
|
Hydrogen-Ion Concentration
|
Concept
|
Adipocytes
|
Concept
|
MAP Kinase Kinase 4
|
Concept
|
Promoter Regions, Genetic
|
Concept
|
Gene Silencing
|
Concept
|
Gene Knockout Techniques
|
Concept
|
Base Sequence
|
Concept
|
Alleles
|
Concept
|
Plasmids
|
Concept
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Genes, Recessive
|
Concept
|
Follow-Up Studies
|
Concept
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Gene Knockdown Techniques
|
Concept
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Embryonic Development
|
Concept
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3-Methyl-2-Oxobutanoate Dehydrogenase (Lipoamide)
|
Concept
|
Genome, Viral
|
Concept
|
Immunohistochemistry
|
Concept
|
Glioblastoma
|
Concept
|
Injections, Intravenous
|
Concept
|
CD36 Antigens
|
Concept
|
Gene Transfer Techniques
|
Concept
|
Cytomegalovirus
|
Concept
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Cell Line, Tumor
|
Concept
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Molecular Sequence Data
|
Concept
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Osmotic Pressure
|
Concept
|
Choroidal Neovascularization
|
Academic Article
|
Prefrontal cortical dysfunction after overexpression of histone deacetylase 1.
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Academic Article
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A single intravenous rAAV injection as late as P20 achieves efficacious and sustained CNS Gene therapy in Canavan mice.
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Academic Article
|
Dicer expression is essential for adult midbrain dopaminergic neuron maintenance and survival.
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Academic Article
|
The potential of adeno-associated viral vectors for gene delivery to muscle tissue.
|
Academic Article
|
Global CNS transduction of adult mice by intravenously delivered rAAVrh.8 and rAAVrh.10 and nonhuman primates by rAAVrh.10.
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Academic Article
|
State-of-the-art human gene therapy: part I. Gene delivery technologies.
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Academic Article
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The PPARa-FGF21 hormone axis contributes to metabolic regulation by the hepatic JNK signaling pathway.
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Academic Article
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State-of-the-art human gene therapy: part II. Gene therapy strategies and clinical applications.
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Academic Article
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Adeno-associated virus-mediated microRNA delivery and therapeutics.
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Academic Article
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Increased CRF signalling in a ventral tegmental area-interpeduncular nucleus-medial habenula circuit induces anxiety during nicotine withdrawal.
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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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Adenovirus-Mediated Somatic Genome Editing of Pten by CRISPR/Cas9 in Mouse Liver in Spite of Cas9-Specific Immune Responses.
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Academic Article
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Making the White Matter Matters: Progress in Understanding Canavan's Disease and Therapeutic Interventions Through Eight Decades.
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Academic Article
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Gene Therapy for the Treatment of Neurological Disorders: Metabolic Disorders.
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Academic Article
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Efficient and Targeted Transduction of Nonhuman Primate Liver With Systemically Delivered Optimized AAV3B Vectors.
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Academic Article
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Cutting Edge: DNA in the Lung Microenvironment during Influenza Virus Infection Tempers Inflammation by Engaging the DNA Sensor AIM2.
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Academic Article
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Adeno-associated Virus Serotype Vectors Efficiently Transduce Normal Prostate Tissue and Prostate Cancer Cells.
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Academic Article
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Therapeutic rAAVrh10 Mediated SOD1 Silencing in Adult SOD1(G93A) Mice and Nonhuman Primates.
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Academic Article
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Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector.
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Academic Article
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rAAV Gene Therapy in a Canavan's Disease Mouse Model Reveals Immune Impairments and an Extended Pathology Beyond the Central Nervous System.
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Academic Article
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Systemic AAV9-IFN? gene delivery treats highly invasive glioblastoma.
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Academic Article
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Empty Virions In AAV8 Vector Preparations Reduce Transduction Efficiency And May Cause Total Viral Particle Dose-Limiting Side-Effects.
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Academic Article
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Examination of the Blood Brain Barrier integrity in a mouse model of the neurodegenerative Canavan's disease.
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Academic Article
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Intracranial AAV-IFN-? gene therapy eliminates invasive xenograft glioblastoma and improves survival in orthotopic syngeneic murine model.
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Academic Article
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The Sodium Channel ?4 Auxiliary Subunit Selectively Controls Long-Term Depression in Core Nucleus Accumbens Medium Spiny Neurons.
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Academic Article
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Fat mass and obesity-associated protein attenuates lipid accumulation in macrophage foam cells and alleviates atherosclerosis in apolipoprotein E-deficient mice.
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Academic Article
|
Rod Outer Segment Development Influences AAV-Mediated Photoreceptor Transduction After Subretinal Injection.
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Academic Article
|
Short DNA Hairpins Compromise Recombinant Adeno-Associated Virus Genome Homogeneity.
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Academic Article
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A Rationally Engineered Capsid Variant of AAV9 for Systemic CNS-Directed and Peripheral Tissue-Detargeted Gene Delivery in Neonates.
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Academic Article
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Adeno-associated Virus Genome Population Sequencing Achieves Full Vector Genome Resolution and Reveals Human-Vector Chimeras.
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Academic Article
|
MicroRNA 122, Regulated by GRLH2, Protects Livers of Mice and?Patients From Ethanol-Induced Liver Disease.
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Academic Article
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Cas9-mediated allelic exchange repairs compound heterozygous recessive mutations in mice.
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Academic Article
|
In Vivo Genome Editing Partially Restores Alpha1-Antitrypsin in a Murine Model of AAT Deficiency.
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Academic Article
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Slow Infusion of Recombinant Adeno-Associated Viruses into the Mouse Cerebrospinal Fluid Space.
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Academic Article
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Streamlined ex vivo and in vivo genome editing in mouse embryos using recombinant adeno-associated viruses.
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Academic Article
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Taking a Hint from Structural Biology: To Better Understand AAV Transport across the BBB.
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Academic Article
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Adeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene Transfer.
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Academic Article
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Artificial miRNAs Reduce Human Mutant Huntingtin Throughout the Striatum in a Transgenic Sheep Model of Huntington's Disease.
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Academic Article
|
Transcriptome Profiling of Neovascularized Corneas Reveals miR-204 as a Multi-target Biotherapy Deliverable by rAAVs.
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Academic Article
|
Selective Neuronal Uptake and Distribution of AAVrh8, AAV9, and AAVrh10 in Sheep After Intra-Striatal Administration.
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Academic Article
|
Gene Therapy Using a miniCEP290 Fragment Delays Photoreceptor Degeneration in a Mouse Model of Leber Congenital Amaurosis.
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Academic Article
|
Evaluation of portable colposcopy and human papillomavirus testing for screening of cervical cancer in rural China.
|
Academic Article
|
Adeno-associated virus vector as a platform for gene therapy delivery.
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Academic Article
|
Intravenous Infusion of AAV for Widespread Gene Delivery to the Nervous System.
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Academic Article
|
High-Throughput Quantification of In Vivo Adeno-Associated Virus Transduction with Barcoded Non-Coding RNAs.
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Academic Article
|
Bone-targeting AAV-mediated silencing of Schnurri-3 prevents bone loss in osteoporosis.
|
Academic Article
|
Conditional, inducible gene silencing in dopamine neurons reveals a sex-specific role for Rit2 GTPase in acute cocaine response and striatal function.
|
Academic Article
|
Gene Delivery to Nonhuman Primate Preimplantation Embryos Using Recombinant Adeno-Associated Virus.
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Academic Article
|
Effective and Accurate Gene Silencing by a Recombinant AAV-Compatible MicroRNA Scaffold.
|
Academic Article
|
Targeted Complement Inhibition at Synapses Prevents Microglial Synaptic Engulfment and Synapse Loss in Demyelinating Disease.
|
Academic Article
|
CRISPR-Based Therapeutic Genome Editing: Strategies and In?Vivo Delivery by AAV Vectors.
|
Academic Article
|
Bone-Targeting AAV-Mediated Gene Silencing in Osteoclasts for Osteoporosis Therapy.
|
Academic Article
|
Structural characterization of a novel human adeno-associated virus capsid with neurotropic properties.
|
Academic Article
|
AAV-Genome Population Sequencing of Vectors Packaging CRISPR Components Reveals Design-Influenced Heterogeneity.
|
Academic Article
|
Is smaller better? Vaccine targeting recombinant receptor-binding domain might hold the key for mass production of effective prophylactics to fight the COVID-19 pandemic.
|
Academic Article
|
Two-Plasmid Packaging System for Recombinant Adeno-Associated Virus.
|
Academic Article
|
A feed-forward regulatory loop in adipose tissue promotes signaling by the hepatokine FGF21.
|
Academic Article
|
AAV-Mediated Gene Therapy for Glycosphingolipid Biosynthesis Deficiencies.
|
Academic Article
|
Viral vector platforms within the gene therapy landscape.
|
Academic Article
|
Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice.
|
Academic Article
|
Canavan Disease as a Model for Gene Therapy-Mediated Myelin Repair.
|
Academic Article
|
Novel Combinatorial MicroRNA-Binding Sites in AAV Vectors Synergistically Diminish Antigen Presentation and Transgene Immunity for Efficient and Stable Transduction.
|
Academic Article
|
Vectored Immunotherapeutics for Infectious Diseases: Can rAAVs Be The Game Changers for Fighting Transmissible Pathogens?
|
Academic Article
|
Low-Dose Recombinant Adeno-Associated Virus-Mediated Inhibition of Vascular Endothelial Growth Factor Can Treat Neovascular Pathologies Without Inducing Retinal Vasculitis.
|
Academic Article
|
AAV5 delivery of CRISPR-Cas9 supports effective genome editing in mouse lung airway.
|
Academic Article
|
Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo.
|
Academic Article
|
Modulating immune responses to AAV by expanded polyclonal T-regs and capsid specific chimeric antigen receptor T-regulatory cells.
|
Concept
|
Alpha-Ketoglutarate-Dependent Dioxygenase FTO
|
Concept
|
Gene Editing
|
Concept
|
Memory, Long-Term
|
Concept
|
HEK293 Cells
|
Concept
|
Infusions, Intraventricular
|
Concept
|
High-Throughput Nucleotide Sequencing
|
Concept
|
Diet, High-Fat
|
Concept
|
Nonsense Mediated mRNA Decay
|
Concept
|
Transcriptome
|
Concept
|
Plaque, Atherosclerotic
|
Concept
|
Intravitreal Injections
|
Concept
|
Pandemics
|
Concept
|
Cellular Microenvironment
|
Concept
|
Real-Time Polymerase Chain Reaction
|
Concept
|
Non-alcoholic Fatty Liver Disease
|
Concept
|
DNA End-Joining Repair
|
Concept
|
Recombinational DNA Repair
|
Concept
|
Infusions, Spinal
|
Concept
|
Clustered Regularly Interspaced Short Palindromic Repeats
|
Concept
|
CRISPR-Cas Systems
|
Concept
|
ATP Binding Cassette Transporter 1
|
Concept
|
Interpeduncular Nucleus
|
Academic Article
|
Regulation of sclerostin by the SIRT1 stabilization pathway in osteocytes.
|
Academic Article
|
Human and Insect Cell-Produced Recombinant Adeno-Associated Viruses Show Differences in Genome Heterogeneity.
|
Academic Article
|
AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice.
|
Academic Article
|
Durability of transgene expression after rAAV gene therapy.
|
Academic Article
|
Adenine Base Editing In Vivo with a Single Adeno-Associated Virus Vector.
|
Academic Article
|
Direct ITR-to-ITR Nanopore Sequencing of AAV Vector Genomes.
|
Academic Article
|
Impaired mitochondrial oxidative metabolism in skeletal progenitor cells leads to musculoskeletal disintegration.
|
Academic Article
|
Suppression of heterotopic ossification in fibrodysplasia ossificans progressiva using AAV gene delivery.
|
Academic Article
|
AAV-mediated delivery of osteoblast/osteoclast-regulating miRNAs for osteoporosis therapy.
|
Academic Article
|
Paradoxical activation of transcription factor SREBP1c and de novo lipogenesis by hepatocyte-selective ATP-citrate lyase depletion in obese mice.
|
Academic Article
|
Ocular Drug Delivery: Advancements and Innovations.
|
Academic Article
|
WNT-modulating gene silencers as a gene therapy for osteoporosis, bone fracture, and critical-sized bone defects.
|
Academic Article
|
Divalent siRNAs are bioavailable in the lung and efficiently block SARS-CoV-2 infection.
|
Academic Article
|
Self-delivering CRISPR RNAs for AAV Co-delivery and Genome Editing in vivo.
|
Academic Article
|
Schnurri-3 inhibition suppresses bone and joint damage in models of rheumatoid arthritis.
|
Academic Article
|
Engineering Nme2Cas9 Adenine Base Editors with Improved Activity and Targeting Scope.
|
Academic Article
|
Renewal of oligodendrocyte lineage reverses dysmyelination and CNS neurodegeneration through corrected N-acetylaspartate metabolism.
|
Academic Article
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Querying Recombination Junctions of Replication-Competent Adeno-Associated Viruses in Gene Therapy Vector Preparations with Single Molecule, Real-Time Sequencing.
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AAV-Mediated Targeting of the Activin A-ACVR1R206H Signaling in Fibrodysplasia Ossificans Progressiva.
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Non-canonical amino acid incorporation into AAV5 capsid enhances lung transduction in mice.
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Self-delivering, chemically modified CRISPR RNAs for AAV co-delivery and genome editing in vivo.
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A Fluorescent Reporter Mouse for In Vivo Assessment of Genome Editing with Diverse Cas Nucleases and Prime Editors.
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AAV-based gene editing of type 1 collagen mutation to treat osteogenesis imperfecta.
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Domain-inlaid Nme2Cas9 adenine base editors with improved activity and targeting scope.
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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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Development of AAV-Mediated Gene Therapy Approaches to Treat Skeletal Diseases.
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Producing high-quantity and high-quality recombinant adeno-associated virus by low-cis triple transfection.
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Adeno-associated virus as a delivery vector for gene therapy of human diseases.
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Advances in Bone-Targeting Drug Delivery: Emerging Strategies Using Adeno-Associated Virus.
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AAV5 Delivery of CRISPR/Cas9 Mediates Genome Editing in the Lungs of Young Rhesus Monkeys.
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Engineering a targeted and safe bone anabolic gene therapy to treat osteoporosis in alveolar bone loss.
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Developing AAV-delivered nonsense suppressor tRNAs for neurological disorders.
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Systemic gene therapy corrects the neurological phenotype in a mouse model of NGLY1 deficiency.
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The AAV2.7m8 capsid packages a higher degree of heterogeneous vector genomes than AAV2.
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Academic Article
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Recombinant adeno-associated virus as a delivery platform for ocular gene therapy: A comprehensive review.
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Academic Article
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Rhodoquinone carries electrons in the mammalian electron transport chain.
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Academic Article
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Comprehensive Review of Osteogenesis Imperfecta: Current Treatments and Future Innovations.
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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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