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Subject Areas on Research
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A CRISPR Screen Identifies the Cell Polarity Determinant Crumbs 3 as an Adeno-associated Virus Restriction Factor in Hepatocytes.
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A small regulatory element from chromosome 19 enhances liver-specific gene expression.
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AAV CRISPR editing rescues cardiac and muscle function for 18 months in dystrophic mice.
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AAV Gene Therapy for MPS1-associated Corneal Blindness.
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AAV exploits subcellular stress associated with inflammation, endoplasmic reticulum expansion, and misfolded proteins in models of cystic fibrosis.
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AAV vector-mediated reversal of hypoglycemia in canine and murine glycogen storage disease type Ia.
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AAV9 Edits Muscle Stem Cells in Normal and Dystrophic Adult Mice.
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AAV9 gene replacement therapy for respiratory insufficiency in very-long chain acyl-CoA dehydrogenase deficiency.
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Active immunization with tumor cells transduced by a novel AAV plasmid-based gene delivery system.
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Active immunotherapy with transiently transfected cytokine-secreting tumor cells inhibits breast cancer metastases in tumor-bearing animals.
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Adeno-Associated Viral Vector (Serotype 2)-Nerve Growth Factor for Patients With Alzheimer Disease: A Randomized Clinical Trial.
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Adeno-Associated Virus Capsid-Promoter Interactions in the Brain Translate from Rat to the Nonhuman Primate.
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Adeno-Associated Virus-Mediated Gene Therapy in the Mashlool, Atp1a3Mashl/+, Mouse Model of Alternating Hemiplegia of Childhood.
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Adeno-associated viral vectors based on serotype 3b use components of the fibroblast growth factor receptor signaling complex for efficient transduction.
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Adeno-associated viral vectors: background and technical aspects.
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Adeno-associated virus gene transfer into renal cells: potential for in vivo gene delivery.
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Adeno-associated virus serotypes: vector toolkit for human gene therapy.
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Adeno-associated virus type 2 (AAV2) capsid-specific cytotoxic T lymphocytes eliminate only vector-transduced cells coexpressing the AAV2 capsid in vivo.
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Adeno-associated virus type 2 contains an integrin alpha5beta1 binding domain essential for viral cell entry.
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Adeno-associated virus vectored immunoprophylaxis to prevent HIV in healthy adults: a phase 1 randomised controlled trial.
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Adeno-associated virus vectors: potential applications for cancer gene therapy.
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Adeno-associated virus-delivered artificial microRNA extends survival and delays paralysis in an amyotrophic lateral sclerosis mouse model.
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Adeno-associated virus-mediated ILK gene silencing in the rat NAc core.
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Adeno-associated virus-mediated antiangiogenic gene therapy with thrombospondin-1 type 1 repeats and endostatin.
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Adeno-associated virus-mediated gene therapy for metabolic myopathy.
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Adjunctive β2-agonist treatment reduces glycogen independently of receptor-mediated acid α-glucosidase uptake in the limb muscles of mice with Pompe disease.
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Adjunctive β2-agonists reverse neuromuscular involvement in murine Pompe disease.
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Age-related efficacy with an AAV vector in Fabry disease mice.
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An Adeno-Associated Virus-Based Toolkit for Preferential Targeting and Manipulating Quiescent Neural Stem Cells in the Adult Hippocampus.
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An allele-specific hammerhead ribozyme gene therapy for a porcine model of autosomal dominant retinitis pigmentosa.
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An emerging adeno-associated viral vector pipeline for cardiac gene therapy.
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An siRNA Screen Identifies the U2 snRNP Spliceosome as a Host Restriction Factor for Recombinant Adeno-associated Viruses.
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Antibody formation and mannose-6-phosphate receptor expression impact the efficacy of muscle-specific transgene expression in murine Pompe disease.
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Bacterial CRISPR/Cas DNA endonucleases: A revolutionary technology that could dramatically impact viral research and treatment.
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Baculoviral IAP repeat-containing-4 protects optic nerve axons in a rat glaucoma model.
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Bioluminescent virion shells: new tools for quantitation of AAV vector dynamics in cells and live animals.
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Biophysical and ultrastructural characterization of adeno-associated virus capsid uncoating and genome release.
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Bone marrow transplantation augments the effect of brain- and spinal cord-directed adeno-associated virus 2/5 gene therapy by altering inflammation in the murine model of globoid-cell leukodystrophy.
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Calcineurin activation causes retinal ganglion cell degeneration.
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Calcium upregulation by percutaneous administration of gene therapy in patients with cardiac disease (CUPID 2): a randomised, multinational, double-blind, placebo-controlled, phase 2b trial.
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Cardiac I-1c overexpression with reengineered AAV improves cardiac function in swine ischemic heart failure.
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Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models.
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Cellular immune response to cryptic epitopes during therapeutic gene transfer.
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Cellular transduction mechanisms of adeno-associated viral vectors.
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Characterization of Staphylococcus aureus Cas9: a smaller Cas9 for all-in-one adeno-associated virus delivery and paired nickase applications.
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Characterization of the Adeno-Associated Virus 1 and 6 Sialic Acid Binding Site.
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Chemical Modulation of Endocytic Sorting Augments Adeno-associated Viral Transduction.
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Chronic recurrent myocardial ischemic injury is significantly attenuated by pre-emptive adeno-associated virus heme oxygenase-1 gene delivery.
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Clinical applications involving CNS gene transfer.
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Coevolution of Adeno-associated Virus Capsid Antigenicity and Tropism through a Structure-Guided Approach.
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Complete correction of hyperphenylalaninemia following liver-directed, recombinant AAV2/8 vector-mediated gene therapy in murine phenylketonuria.
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Correction of glycogen storage disease type II by an adeno-associated virus vector containing a muscle-specific promoter.
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Correction of multiple striated muscles in murine Pompe disease through adeno-associated virus-mediated gene therapy.
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Cre-dependent adeno-associated virus preparation and delivery for labeling neurons in the mouse brain.
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Cross-dressing the virion: the transcapsidation of adeno-associated virus serotypes functionally defines subgroups.
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Cytotoxic-T-lymphocyte-mediated elimination of target cells transduced with engineered adeno-associated virus type 2 vector in vivo.
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Defining Transcription Regulatory Elements in the Human Frataxin Gene: Implications for Gene Therapy.
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Delivery of glucose-6-phosphatase in a canine model for glycogen storage disease, type Ia, with adeno-associated virus (AAV) vectors.
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Development of Patient-specific AAV Vectors After Neutralizing Antibody Selection for Enhanced Muscle Gene Transfer.
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Development of rAAV2-CFTR: History of the First rAAV Vector Product to be Used in Humans.
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Differential effects of unfolded protein response pathways on axon injury-induced death of retinal ganglion cells.
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Distinctive gene transduction efficiencies of commonly used viral vectors in the retina.
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Dystrophin immunity in Duchenne's muscular dystrophy.
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Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia.
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Efficacious Androgen Hormone Administration in Combination with Adeno-Associated Virus Vector-Mediated Gene Therapy in Female Mice with Pompe Disease.
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Efficacy of an adeno-associated virus 8-pseudotyped vector in glycogen storage disease type II.
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Efficient gene transfer with adeno-associated virus-based plasmids complexed to cationic liposomes for gene therapy of human prostate cancer.
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Engineering AAV receptor footprints for gene therapy.
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Engineering and Selection of Shuffled AAV Genomes: A New Strategy for Producing Targeted Biological Nanoparticles.
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Engineering liver-detargeted AAV9 vectors for cardiac and musculoskeletal gene transfer.
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Engraftment of a galactose receptor footprint onto adeno-associated viral capsids improves transduction efficiency.
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Enhanced efficacy from gene therapy in Pompe disease using coreceptor blockade.
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Enhanced efficacy of an AAV vector encoding chimeric, highly secreted acid alpha-glucosidase in glycogen storage disease type II.
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Enhanced response to enzyme replacement therapy in Pompe disease after the induction of immune tolerance.
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Enhancement of rAAV2-mediated transgene expression in retina cells in vitro and in vivo by coadministration of low-dose chemotherapeutic drugs.
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Epigenetic Silencing of Recombinant Adeno-associated Virus Genomes by NP220 and the HUSH Complex.
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Evasion of immune responses to introduced human acid alpha-glucosidase by liver-restricted expression in glycogen storage disease type II.
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Extracellular but not cytosolic superoxide dismutase protects against oxidant-mediated endothelial dysfunction.
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Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy.
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Functional analysis of the putative integrin recognition motif on adeno-associated virus 9.
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Gene delivery approaches to heart failure treatment.
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Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial.
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Gene delivery of neurturin to putamen and substantia nigra in Parkinson disease: A double-blind, randomized, controlled trial.
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Gene modification of primary tumor cells for active immunotherapy of human breast and ovarian cancer.
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Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years.
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Gene therapy strategy for long-term myocardial protection using adeno-associated virus-mediated delivery of heme oxygenase gene.
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Generation and characterization of anti-Adeno-associated virus serotype 8 (AAV8) and anti-AAV9 monoclonal antibodies.
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Generation of neutralizing activity against human immunodeficiency virus type 1 in serum by antibody gene transfer.
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Generation of recombinant adeno-associated virus vectors by a complete adenovirus-mediated approach.
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Genetic background influences LRRK2-mediated Rab phosphorylation in the rat brain.
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Glycan binding avidity determines the systemic fate of adeno-associated virus type 9.
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Glymphatic fluid transport controls paravascular clearance of AAV vectors from the brain.
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Hepatorenal correction in murine glycogen storage disease type I with a double-stranded adeno-associated virus vector.
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High-resolution labeling and functional manipulation of specific neuron types in mouse brain by Cre-activated viral gene expression.
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Human carbonic anhydrase-8 AAV8 gene therapy inhibits nerve growth factor signaling producing prolonged analgesia and anti-hyperalgesia in mice.
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Human galectin 3 binding protein interacts with recombinant adeno-associated virus type 6.
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Hydrostatic isolated limb perfusion with adeno-associated virus vectors enhances correction of skeletal muscle in Pompe disease.
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Immunodominant liver-specific expression suppresses transgene-directed immune responses in murine pompe disease.
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Immunomodulatory gene therapy prevents antibody formation and lethal hypersensitivity reactions in murine pompe disease.
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Immunomodulatory, liver depot gene therapy for Pompe disease.
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Impact of Heparan Sulfate Binding on Transduction of Retina by Recombinant Adeno-Associated Virus Vectors.
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Impaired clearance of accumulated lysosomal glycogen in advanced Pompe disease despite high-level vector-mediated transgene expression.
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Improved survival and reduced phenotypic severity following AAV9/MECP2 gene transfer to neonatal and juvenile male Mecp2 knockout mice.
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In Situ Gene Therapy via AAV-CRISPR-Cas9-Mediated Targeted Gene Regulation.
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In search of proof-of-concept: gene therapy for glycogen storage disease type Ia.
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In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy.
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Increased tissue perfusion promotes capillary dysplasia in the ALK1-deficient mouse brain following VEGF stimulation.
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Increased vascular smooth muscle contractility in TRPC6-/- mice.
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Interaction between TRPV1-expressing neurons in the hypothalamus.
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Intra- and inter-subunit disulfide bond formation is nonessential in adeno-associated viral capsids.
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Intracerebral Expression of AAV-APOE4 Is Not Sufficient to Alter Tau Burden in Two Distinct Models of Tauopathy.
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Intracerebral adeno-associated virus gene delivery of apolipoprotein E2 markedly reduces brain amyloid pathology in Alzheimer's disease mouse models.
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Intralingual Administration of AAVrh10-miRSOD1 Improves Respiratory But Not Swallowing Function in a Superoxide Dismutase-1 Mouse Model of Amyotrophic Lateral Sclerosis.
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Intrapleural administration of AAV9 improves neural and cardiorespiratory function in Pompe disease.
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Intrastromal Gene Therapy Prevents and Reverses Advanced Corneal Clouding in a Canine Model of Mucopolysaccharidosis I.
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Intravitreal delivery of a novel AAV vector targets ON bipolar cells and restores visual function in a mouse model of complete congenital stationary night blindness.
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Large animal models and new therapies for glycogen storage disease.
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Leucine-rich Repeat Kinase 2 (LRRK2) Pharmacological Inhibition Abates α-Synuclein Gene-induced Neurodegeneration.
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Long-term complications of glycogen storage disease type Ia in the canine model treated with gene replacement therapy.
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Long-term correction of glycogen storage disease type II with a hybrid Ad-AAV vector.
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Long-term efficacy following readministration of an adeno-associated virus vector in dogs with glycogen storage disease type Ia.
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Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy.
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Lowering circulating apolipoprotein E levels improves aged bone fracture healing.
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Mapping and Engineering Functional Domains of the Assembly-Activating Protein of Adeno-associated Viruses.
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Mapping the Structural Determinants Required for AAVrh.10 Transport across the Blood-Brain Barrier.
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Marker rescue of adeno-associated virus (AAV) capsid mutants: a novel approach for chimeric AAV production.
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Minimum Effective Dose to Achieve Biochemical Correction with Adeno-Associated Virus Vector-Mediated Gene Therapy in Mice with Pompe Disease.
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Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy.
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Modulation of Sialic Acid Dependence Influences the Central Nervous System Transduction Profile of Adeno-associated Viruses.
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Multiple roles for sialylated glycans in determining the cardiopulmonary tropism of adeno-associated virus 4.
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Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia.
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Novel adeno-associated virus vector vaccine restricts replication of simian immunodeficiency virus in macaques.
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Optimization of Meniscus Cell Transduction Using Lentivirus and Adeno-Associated Virus for Gene Editing and Tissue Engineering Applications.
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Optimization of a multiplex CRISPR/Cas system for use as an antiviral therapeutic.
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Optimizing delivery for efficient cardiac reprogramming.
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Packaging of an AAV vector encoding human acid alpha-glucosidase for gene therapy in glycogen storage disease type II with a modified hybrid adenovirus-AAV vector.
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Peptide affinity reagents for AAV capsid recognition and purification.
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Persistent expression of human clotting factor IX from mouse liver after intravenous injection of adeno-associated virus vectors.
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Persistent, therapeutically relevant levels of human granulocyte colony-stimulating factor in mice after systemic delivery of adeno-associated virus vectors.
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Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector.
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Physical positioning markedly enhances brain transduction after intrathecal AAV9 infusion.
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Plug-and-Play Protein Modification Using Homology-Independent Universal Genome Engineering.
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Pompe disease gene therapy.
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Potential for germ line transmission after intramyocardial gene delivery by adeno-associated virus.
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Pre-emptive gene therapy using recombinant adeno-associated virus delivery of extracellular superoxide dismutase protects heart against ischemic reperfusion injury, improves ventricular function and prolongs survival.
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Prevalence of AAV1 neutralizing antibodies and consequences for a clinical trial of gene transfer for advanced heart failure.
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Probing the Link among Genomic Cargo, Contact Mechanics, and Nanoindentation in Recombinant Adeno-Associated Virus 2.
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Production of recombinant adeno-associated viral vectors and use for in vitro and in vivo administration.
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Production of recombinant adeno-associated viral vectors and use in in vitro and in vivo administration.
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Production of recombinant adeno-associated viral vectors for in vitro and in vivo use.
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Production of recombinant adeno-associated viral vectors.
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Production, purification and preliminary X-ray crystallographic studies of adeno-associated virus serotype 9.
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Receptor Switching in Newly Evolved Adeno-associated Viruses.
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Reduction of Autophagic Accumulation in Pompe Disease Mouse Model Following Gene Therapy.
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Reengineered AAV vectors: old dog, new tricks.
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Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle.
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Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase.
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Rescuing AAV gene transfer from neutralizing antibodies with an IgG-degrading enzyme.
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Resolving Adeno-Associated Viral Particle Diversity With Charge Detection Mass Spectrometry.
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Restoring neuronal progranulin reverses deficits in a mouse model of frontotemporal dementia.
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Retrograde gene delivery to hypoglossal motoneurons using adeno-associated virus serotype 9.
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Reversion mutation of cDNA CA8-204 minigene construct produces a truncated functional peptide that regulates calcium release in vitro and produces profound analgesia in vivo.
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Ring finger protein 121 is a potent regulator of adeno-associated viral genome transcription.
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Safety and tolerability of AAV8 delivery of a broadly neutralizing antibody in adults living with HIV: a phase 1, dose-escalation trial.
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Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients.
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Salmeterol enhances the cardiac response to gene therapy in Pompe disease.
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Salmeterol with Liver Depot Gene Therapy Enhances the Skeletal Muscle Response in Murine Pompe Disease.
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Single amino acid changes can influence titer, heparin binding, and tissue tropism in different adeno-associated virus serotypes.
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Single amino acid modification of adeno-associated virus capsid changes transduction and humoral immune profiles.
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Sites of recombinant adeno-associated virus integration.
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Stable inhibition of hepatitis B virus proteins by small interfering RNA expressed from viral vectors.
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Strategies to circumvent humoral immunity to adeno-associated viral vectors.
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Structurally Mapping Antigenic Epitopes of Adeno-associated Virus 9: Development of Antibody Escape Variants.
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Structure-guided evolution of antigenically distinct adeno-associated virus variants for immune evasion.
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Surface loop dynamics in adeno-associated virus capsid assembly.
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Sustained correction of motoneuron histopathology following intramuscular delivery of AAV in pompe mice.
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Sustained expression of human factor VIII in mice using a parvovirus-based vector.
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Synergistic Efficacy from Gene Therapy with Coreceptor Blockade and a β2-Agonist in Murine Pompe Disease.
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Systemic Correction of Murine Glycogen Storage Disease Type IV by an AAV-Mediated Gene Therapy.
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Systemic Delivery of AAVB1-GAA Clears Glycogen and Prolongs Survival in a Mouse Model of Pompe Disease.
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Systemic and Persistent Muscle Gene Expression in Rhesus Monkeys with a Liver De-Targeted Adeno-Associated Virus Vector.
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Systemic gene transfer to skeletal muscle using reengineered AAV vectors.
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Targeting cells with single vectors using multiple-feature Boolean logic.
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Terminal N-linked galactose is the primary receptor for adeno-associated virus 9.
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The AAV vector toolkit: poised at the clinical crossroads.
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The Golgi Calcium ATPase Pump Plays an Essential Role in Adeno-associated Virus Trafficking and Transduction.
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The membrane associated accessory protein is an adeno-associated viral egress factor.
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The upstream enhancer elements of the G6PC promoter are critical for optimal G6PC expression in murine glycogen storage disease type Ia.
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Therapeutic potential of AAV-mediated MMP-3 secretion from corneal endothelium in treating glaucoma.
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Therapeutic rAAVrh10 Mediated SOD1 Silencing in Adult SOD1(G93A) Mice and Nonhuman Primates.
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Three-dimensional tissue-engineered human skeletal muscle model of Pompe disease.
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Transduction of renal cells in vitro and in vivo by adeno-associated virus gene therapy vectors.
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Tyrosine cross-linking reveals interfacial dynamics in adeno-associated viral capsids during infection.
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Unique glycan signatures regulate adeno-associated virus tropism in the developing brain.
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X-linked inhibitor of apoptosis protein-mediated attenuation of apoptosis, using a novel cardiac-enhanced adeno-associated viral vector.
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rAAV-compatible MiniPromoters for restricted expression in the brain and eye.
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Keywords of People