Dependovirus

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  Subject Areas on Research

  • A CRISPR Screen Identifies the Cell Polarity Determinant Crumbs 3 as an Adeno-associated Virus Restriction Factor in Hepatocytes. 
  • A small regulatory element from chromosome 19 enhances liver-specific gene expression. 
  • AAV CRISPR editing rescues cardiac and muscle function for 18 months in dystrophic mice. 
  • AAV Gene Therapy for MPS1-associated Corneal Blindness. 
  • AAV exploits subcellular stress associated with inflammation, endoplasmic reticulum expansion, and misfolded proteins in models of cystic fibrosis. 
  • AAV vector-mediated reversal of hypoglycemia in canine and murine glycogen storage disease type Ia. 
  • AAV9 Edits Muscle Stem Cells in Normal and Dystrophic Adult Mice. 
  • AAV9 gene replacement therapy for respiratory insufficiency in very-long chain acyl-CoA dehydrogenase deficiency. 
  • Active immunization with tumor cells transduced by a novel AAV plasmid-based gene delivery system. 
  • Active immunotherapy with transiently transfected cytokine-secreting tumor cells inhibits breast cancer metastases in tumor-bearing animals. 
  • Adeno-Associated Viral Vector (Serotype 2)-Nerve Growth Factor for Patients With Alzheimer Disease: A Randomized Clinical Trial. 
  • Adeno-Associated Virus Capsid-Promoter Interactions in the Brain Translate from Rat to the Nonhuman Primate. 
  • Adeno-Associated Virus-Mediated Gene Therapy in the Mashlool, Atp1a3Mashl/+, Mouse Model of Alternating Hemiplegia of Childhood. 
  • Adeno-associated viral vectors based on serotype 3b use components of the fibroblast growth factor receptor signaling complex for efficient transduction. 
  • Adeno-associated viral vectors: background and technical aspects. 
  • Adeno-associated virus gene transfer into renal cells: potential for in vivo gene delivery. 
  • Adeno-associated virus serotypes: vector toolkit for human gene therapy. 
  • Adeno-associated virus type 2 (AAV2) capsid-specific cytotoxic T lymphocytes eliminate only vector-transduced cells coexpressing the AAV2 capsid in vivo. 
  • Adeno-associated virus type 2 contains an integrin alpha5beta1 binding domain essential for viral cell entry. 
  • Adeno-associated virus vectored immunoprophylaxis to prevent HIV in healthy adults: a phase 1 randomised controlled trial. 
  • Adeno-associated virus vectors: potential applications for cancer gene therapy. 
  • Adeno-associated virus-delivered artificial microRNA extends survival and delays paralysis in an amyotrophic lateral sclerosis mouse model. 
  • Adeno-associated virus-mediated ILK gene silencing in the rat NAc core. 
  • Adeno-associated virus-mediated antiangiogenic gene therapy with thrombospondin-1 type 1 repeats and endostatin. 
  • Adeno-associated virus-mediated gene therapy for metabolic myopathy. 
  • Adjunctive β2-agonist treatment reduces glycogen independently of receptor-mediated acid α-glucosidase uptake in the limb muscles of mice with Pompe disease. 
  • Adjunctive β2-agonists reverse neuromuscular involvement in murine Pompe disease. 
  • Age-related efficacy with an AAV vector in Fabry disease mice. 
  • An Adeno-Associated Virus-Based Toolkit for Preferential Targeting and Manipulating Quiescent Neural Stem Cells in the Adult Hippocampus. 
  • An allele-specific hammerhead ribozyme gene therapy for a porcine model of autosomal dominant retinitis pigmentosa. 
  • An emerging adeno-associated viral vector pipeline for cardiac gene therapy. 
  • An evolved AAV variant enables efficient genetic engineering of murine T cells. 
  • An siRNA Screen Identifies the U2 snRNP Spliceosome as a Host Restriction Factor for Recombinant Adeno-associated Viruses. 
  • Antibody formation and mannose-6-phosphate receptor expression impact the efficacy of muscle-specific transgene expression in murine Pompe disease. 
  • Bacterial CRISPR/Cas DNA endonucleases: A revolutionary technology that could dramatically impact viral research and treatment. 
  • Baculoviral IAP repeat-containing-4 protects optic nerve axons in a rat glaucoma model. 
  • Bioluminescent virion shells: new tools for quantitation of AAV vector dynamics in cells and live animals. 
  • Biophysical and ultrastructural characterization of adeno-associated virus capsid uncoating and genome release. 
  • 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. 
  • Calcineurin activation causes retinal ganglion cell degeneration. 
  • 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. 
  • Cardiac I-1c overexpression with reengineered AAV improves cardiac function in swine ischemic heart failure. 
  • Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models. 
  • Cellular immune response to cryptic epitopes during therapeutic gene transfer. 
  • Cellular transduction mechanisms of adeno-associated viral vectors. 
  • Characterization of Staphylococcus aureus Cas9: a smaller Cas9 for all-in-one adeno-associated virus delivery and paired nickase applications. 
  • Characterization of the Adeno-Associated Virus 1 and 6 Sialic Acid Binding Site. 
  • Chemical Modulation of Endocytic Sorting Augments Adeno-associated Viral Transduction. 
  • Chronic recurrent myocardial ischemic injury is significantly attenuated by pre-emptive adeno-associated virus heme oxygenase-1 gene delivery. 
  • Chronic suppression of heart-failure progression by a pseudophosphorylated mutant of phospholamban via in vivo cardiac rAAV gene delivery. 
  • Clinical applications involving CNS gene transfer. 
  • Coevolution of Adeno-associated Virus Capsid Antigenicity and Tropism through a Structure-Guided Approach. 
  • Complete correction of hyperphenylalaninemia following liver-directed, recombinant AAV2/8 vector-mediated gene therapy in murine phenylketonuria. 
  • Correction of glycogen storage disease type II by an adeno-associated virus vector containing a muscle-specific promoter. 
  • Correction of multiple striated muscles in murine Pompe disease through adeno-associated virus-mediated gene therapy. 
  • Cre-dependent adeno-associated virus preparation and delivery for labeling neurons in the mouse brain. 
  • Cross-dressing the virion: the transcapsidation of adeno-associated virus serotypes functionally defines subgroups. 
  • Cross-species evolution of a highly potent AAV variant for therapeutic gene transfer and genome editing. 
  • Cytotoxic-T-lymphocyte-mediated elimination of target cells transduced with engineered adeno-associated virus type 2 vector in vivo. 
  • Defining Transcription Regulatory Elements in the Human Frataxin Gene: Implications for Gene Therapy. 
  • Delivery of glucose-6-phosphatase in a canine model for glycogen storage disease, type Ia, with adeno-associated virus (AAV) vectors. 
  • Development of Patient-specific AAV Vectors After Neutralizing Antibody Selection for Enhanced Muscle Gene Transfer. 
  • Development of rAAV2-CFTR: History of the First rAAV Vector Product to be Used in Humans. 
  • Differential effects of unfolded protein response pathways on axon injury-induced death of retinal ganglion cells. 
  • Distinctive gene transduction efficiencies of commonly used viral vectors in the retina. 
  • Dystrophin immunity in Duchenne's muscular dystrophy. 
  • Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia. 
  • Efficacious Androgen Hormone Administration in Combination with Adeno-Associated Virus Vector-Mediated Gene Therapy in Female Mice with Pompe Disease. 
  • Efficacy of an adeno-associated virus 8-pseudotyped vector in glycogen storage disease type II. 
  • Efficient gene transfer with adeno-associated virus-based plasmids complexed to cationic liposomes for gene therapy of human prostate cancer. 
  • Engineering AAV receptor footprints for gene therapy. 
  • Engineering and Selection of Shuffled AAV Genomes: A New Strategy for Producing Targeted Biological Nanoparticles. 
  • Engineering liver-detargeted AAV9 vectors for cardiac and musculoskeletal gene transfer. 
  • Engraftment of a galactose receptor footprint onto adeno-associated viral capsids improves transduction efficiency. 
  • Enhanced efficacy from gene therapy in Pompe disease using coreceptor blockade. 
  • Enhanced efficacy of an AAV vector encoding chimeric, highly secreted acid alpha-glucosidase in glycogen storage disease type II. 
  • Enhanced response to enzyme replacement therapy in Pompe disease after the induction of immune tolerance. 
  • Enhancement of rAAV2-mediated transgene expression in retina cells in vitro and in vivo by coadministration of low-dose chemotherapeutic drugs. 
  • Epigenetic Silencing of Recombinant Adeno-associated Virus Genomes by NP220 and the HUSH Complex. 
  • Evasion of immune responses to introduced human acid alpha-glucosidase by liver-restricted expression in glycogen storage disease type II. 
  • Extracellular but not cytosolic superoxide dismutase protects against oxidant-mediated endothelial dysfunction. 
  • Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy. 
  • Functional analysis of the putative integrin recognition motif on adeno-associated virus 9. 
  • Gene delivery approaches to heart failure treatment. 
  • Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial. 
  • Gene delivery of neurturin to putamen and substantia nigra in Parkinson disease: A double-blind, randomized, controlled trial. 
  • Gene modification of primary tumor cells for active immunotherapy of human breast and ovarian cancer. 
  • Gene therapy beyond luxturna: a new horizon of the treatment for inherited retinal disease. 
  • Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years. 
  • Gene therapy strategy for long-term myocardial protection using adeno-associated virus-mediated delivery of heme oxygenase gene. 
  • Generation and characterization of anti-Adeno-associated virus serotype 8 (AAV8) and anti-AAV9 monoclonal antibodies. 
  • Generation of neutralizing activity against human immunodeficiency virus type 1 in serum by antibody gene transfer. 
  • Generation of recombinant adeno-associated virus vectors by a complete adenovirus-mediated approach. 
  • Genetic background influences LRRK2-mediated Rab phosphorylation in the rat brain. 
  • Glycan binding avidity determines the systemic fate of adeno-associated virus type 9. 
  • Glymphatic fluid transport controls paravascular clearance of AAV vectors from the brain. 
  • Hepatorenal correction in murine glycogen storage disease type I with a double-stranded adeno-associated virus vector. 
  • High-resolution labeling and functional manipulation of specific neuron types in mouse brain by Cre-activated viral gene expression. 
  • Human carbonic anhydrase-8 AAV8 gene therapy inhibits nerve growth factor signaling producing prolonged analgesia and anti-hyperalgesia in mice. 
  • Human galectin 3 binding protein interacts with recombinant adeno-associated virus type 6. 
  • Hydrostatic isolated limb perfusion with adeno-associated virus vectors enhances correction of skeletal muscle in Pompe disease. 
  • Immunodominant liver-specific expression suppresses transgene-directed immune responses in murine pompe disease. 
  • Immunomodulatory gene therapy prevents antibody formation and lethal hypersensitivity reactions in murine pompe disease. 
  • Immunomodulatory, liver depot gene therapy for Pompe disease. 
  • Impaired clearance of accumulated lysosomal glycogen in advanced Pompe disease despite high-level vector-mediated transgene expression. 
  • Improved survival and reduced phenotypic severity following AAV9/MECP2 gene transfer to neonatal and juvenile male Mecp2 knockout mice. 
  • In Situ Gene Therapy via AAV-CRISPR-Cas9-Mediated Targeted Gene Regulation. 
  • In search of proof-of-concept: gene therapy for glycogen storage disease type Ia. 
  • In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy. 
  • Increased tissue perfusion promotes capillary dysplasia in the ALK1-deficient mouse brain following VEGF stimulation. 
  • Increased vascular smooth muscle contractility in TRPC6-/- mice. 
  • Interaction between TRPV1-expressing neurons in the hypothalamus. 
  • Intra- and inter-subunit disulfide bond formation is nonessential in adeno-associated viral capsids. 
  • Intracerebral Expression of AAV-APOE4 Is Not Sufficient to Alter Tau Burden in Two Distinct Models of Tauopathy. 
  • Intracerebral adeno-associated virus gene delivery of apolipoprotein E2 markedly reduces brain amyloid pathology in Alzheimer's disease mouse models. 
  • Intralingual Administration of AAVrh10-miRSOD1 Improves Respiratory But Not Swallowing Function in a Superoxide Dismutase-1 Mouse Model of Amyotrophic Lateral Sclerosis. 
  • Intrapleural administration of AAV9 improves neural and cardiorespiratory function in Pompe disease. 
  • Intrastromal Gene Therapy Prevents and Reverses Advanced Corneal Clouding in a Canine Model of Mucopolysaccharidosis I. 
  • Intravitreal Delivery of rAAV2tYF-CB-hRS1 Vector for Gene Augmentation Therapy in Patients with X-Linked Retinoschisis: 1-Year Clinical Results. 
  • Large animal models and new therapies for glycogen storage disease. 
  • Leucine-rich Repeat Kinase 2 (LRRK2) Pharmacological Inhibition Abates α-Synuclein Gene-induced Neurodegeneration. 
  • Long-term complications of glycogen storage disease type Ia in the canine model treated with gene replacement therapy. 
  • Long-term correction of glycogen storage disease type II with a hybrid Ad-AAV vector. 
  • Long-term efficacy following readministration of an adeno-associated virus vector in dogs with glycogen storage disease type Ia. 
  • Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy. 
  • Lowering circulating apolipoprotein E levels improves aged bone fracture healing. 
  • Mapping and Engineering Functional Domains of the Assembly-Activating Protein of Adeno-associated Viruses. 
  • Mapping the Structural Determinants Required for AAVrh.10 Transport across the Blood-Brain Barrier. 
  • Marker rescue of adeno-associated virus (AAV) capsid mutants: a novel approach for chimeric AAV production. 
  • Minimum Effective Dose to Achieve Biochemical Correction with Adeno-Associated Virus Vector-Mediated Gene Therapy in Mice with Pompe Disease. 
  • Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy. 
  • Modulation of Sialic Acid Dependence Influences the Central Nervous System Transduction Profile of Adeno-associated Viruses. 
  • Multiple roles for sialylated glycans in determining the cardiopulmonary tropism of adeno-associated virus 4. 
  • Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia. 
  • Novel adeno-associated virus vector vaccine restricts replication of simian immunodeficiency virus in macaques. 
  • Optimization of Meniscus Cell Transduction Using Lentivirus and Adeno-Associated Virus for Gene Editing and Tissue Engineering Applications. 
  • Optimization of a multiplex CRISPR/Cas system for use as an antiviral therapeutic. 
  • Optimizing delivery for efficient cardiac reprogramming. 
  • 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. 
  • Peptide affinity reagents for AAV capsid recognition and purification. 
  • Persistent expression of human clotting factor IX from mouse liver after intravenous injection of adeno-associated virus vectors. 
  • Persistent, therapeutically relevant levels of human granulocyte colony-stimulating factor in mice after systemic delivery of adeno-associated virus vectors. 
  • Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector. 
  • Physical positioning markedly enhances brain transduction after intrathecal AAV9 infusion. 
  • Plug-and-Play Protein Modification Using Homology-Independent Universal Genome Engineering. 
  • Pompe disease gene therapy. 
  • Potential for germ line transmission after intramyocardial gene delivery by adeno-associated virus. 
  • 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. 
  • Prevalence of AAV1 neutralizing antibodies and consequences for a clinical trial of gene transfer for advanced heart failure. 
  • Probing the Link among Genomic Cargo, Contact Mechanics, and Nanoindentation in Recombinant Adeno-Associated Virus 2. 
  • Production of recombinant adeno-associated viral vectors and use for in vitro and in vivo administration. 
  • Production of recombinant adeno-associated viral vectors and use in in vitro and in vivo administration. 
  • Production of recombinant adeno-associated viral vectors for in vitro and in vivo use. 
  • Production of recombinant adeno-associated viral vectors. 
  • Production, purification and preliminary X-ray crystallographic studies of adeno-associated virus serotype 9. 
  • Receptor Switching in Newly Evolved Adeno-associated Viruses. 
  • Reduction of Autophagic Accumulation in Pompe Disease Mouse Model Following Gene Therapy. 
  • Reengineered AAV vectors: old dog, new tricks. 
  • Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle. 
  • Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase. 
  • Rescuing AAV gene transfer from neutralizing antibodies with an IgG-degrading enzyme. 
  • Resolving Adeno-Associated Viral Particle Diversity With Charge Detection Mass Spectrometry. 
  • Restoring neuronal progranulin reverses deficits in a mouse model of frontotemporal dementia. 
  • Retrograde gene delivery to hypoglossal motoneurons using adeno-associated virus serotype 9. 
  • 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. 
  • Ring finger protein 121 is a potent regulator of adeno-associated viral genome transcription. 
  • Robust adenoviral and adeno-associated viral gene transfer to the in vivo murine heart: application to study of phospholamban physiology. 
  • Safety and tolerability of AAV8 delivery of a broadly neutralizing antibody in adults living with HIV: a phase 1, dose-escalation trial. 
  • Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients. 
  • Salmeterol enhances the cardiac response to gene therapy in Pompe disease. 
  • Salmeterol with Liver Depot Gene Therapy Enhances the Skeletal Muscle Response in Murine Pompe Disease. 
  • Single amino acid changes can influence titer, heparin binding, and tissue tropism in different adeno-associated virus serotypes. 
  • Single amino acid modification of adeno-associated virus capsid changes transduction and humoral immune profiles. 
  • Sites of recombinant adeno-associated virus integration. 
  • Stable inhibition of hepatitis B virus proteins by small interfering RNA expressed from viral vectors. 
  • Strategies to circumvent humoral immunity to adeno-associated viral vectors. 
  • Structurally Mapping Antigenic Epitopes of Adeno-associated Virus 9: Development of Antibody Escape Variants. 
  • Structure-guided evolution of antigenically distinct adeno-associated virus variants for immune evasion. 
  • Surface loop dynamics in adeno-associated virus capsid assembly. 
  • Sustained correction of motoneuron histopathology following intramuscular delivery of AAV in pompe mice. 
  • Sustained expression of human factor VIII in mice using a parvovirus-based vector. 
  • Synergistic Efficacy from Gene Therapy with Coreceptor Blockade and a β2-Agonist in Murine Pompe Disease. 
  • Systemic Correction of Murine Glycogen Storage Disease Type IV by an AAV-Mediated Gene Therapy. 
  • Systemic Delivery of AAVB1-GAA Clears Glycogen and Prolongs Survival in a Mouse Model of Pompe Disease. 
  • Systemic and Persistent Muscle Gene Expression in Rhesus Monkeys with a Liver De-Targeted Adeno-Associated Virus Vector. 
  • Systemic gene transfer to skeletal muscle using reengineered AAV vectors. 
  • Targeting cells with single vectors using multiple-feature Boolean logic. 
  • Terminal N-linked galactose is the primary receptor for adeno-associated virus 9. 
  • The AAV vector toolkit: poised at the clinical crossroads. 
  • The Golgi Calcium ATPase Pump Plays an Essential Role in Adeno-associated Virus Trafficking and Transduction. 
  • The membrane associated accessory protein is an adeno-associated viral egress factor. 
  • The upstream enhancer elements of the G6PC promoter are critical for optimal G6PC expression in murine glycogen storage disease type Ia. 
  • Therapeutic potential of AAV-mediated MMP-3 secretion from corneal endothelium in treating glaucoma. 
  • Therapeutic rAAVrh10 Mediated SOD1 Silencing in Adult SOD1(G93A) Mice and Nonhuman Primates. 
  • Three-dimensional tissue-engineered human skeletal muscle model of Pompe disease. 
  • Transduction of renal cells in vitro and in vivo by adeno-associated virus gene therapy vectors. 
  • Tyrosine cross-linking reveals interfacial dynamics in adeno-associated viral capsids during infection. 
  • Unique glycan signatures regulate adeno-associated virus tropism in the developing brain. 
  • Viral sequences enable efficient and tissue-specific expression of transgenes in Xenopus. 
  • X-linked inhibitor of apoptosis protein-mediated attenuation of apoptosis, using a novel cardiac-enhanced adeno-associated viral vector. 
  • rAAV-compatible MiniPromoters for restricted expression in the brain and eye. 
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  Keywords of People

  • Milano, Carmelo Alessio, Joseph W. and Dorothy W. Beard Distinguished Professor of Experimental Surgery, Surgery, Cardiovascular and Thoracic Surgery
  • Turner, Dennis Alan, Professor of Neurosurgery, Biomedical Engineering