The role of the adeno-associated virus capsid in gene transfer (Q37120438)

18 August 2017

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title

The role of the adeno-associated virus capsid in gene transfer (English)

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18 August 2017

Mavis Agbandje-McKenna

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4

object named as

Mavis Agbandje-McKenna

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18 August 2017

2

Veronique Blouin

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18 August 2017

Kim M Van Vliet

1

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18 August 2017

Nicole Brument

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3

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18 August 2017

Richard O Snyder

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5

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18 August 2017

language of work or name

English

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publication date

1 January 2008

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Methods in Molecular Biology

18 August 2017

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18 August 2017

437

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18 August 2017

51-91

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Neonatal intramuscular injection with recombinant adeno-associated virus results in prolonged beta-glucuronidase expression in situ and correction of liver pathology in mucopolysaccharidosis type VII mice.

18 August 2017

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12 December 2020

Repeated adeno-associated virus serotype 2 aerosol-mediated cystic fibrosis transmembrane regulator gene transfer to the lungs of patients with cystic fibrosis: a multicenter, double-blind, placebo-controlled trial

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12 December 2020

Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5, and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate models

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12 December 2020

A phase I/II study of tgAAV-CF for the treatment of chronic sinusitis in patients with cystic fibrosis

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12 December 2020

A phase I study of an adeno-associated virus-CFTR gene vector in adult CF patients with mild lung disease

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12 December 2020

AAV2/5-mediated NGF gene delivery protects septal cholinergic neurons following axotomy

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12 December 2020

Proteolytic mapping of the adeno-associated virus capsid

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12 December 2020

A novel recombinant adeno-associated virus vaccine reduces behavioral impairment and beta-amyloid plaques in a mouse model of Alzheimer's disease

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12 December 2020

Production, purification, crystallization and preliminary X-ray structural studies of adeno-associated virus serotype 5

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12 December 2020

Assembly of viruslike particles by recombinant structural proteins of adeno-associated virus type 2 in insect cells

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12 December 2020

Regulated expression of erythropoietin from an AAV vector safely improves the anemia of beta-thalassemia in a mouse model.

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12 December 2020

Adeno-associated virus type 5 (AAV5) but not AAV2 binds to the apical surfaces of airway epithelia and facilitates gene transfer

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12 December 2020

Enhanced gene transfer efficiency in the murine striatum and an orthotopic glioblastoma tumor model, using AAV-7- and AAV-8-pseudotyped vectors

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12 December 2020

A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies.

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12 December 2020

Cardiomyocyte-specific gene expression following recombinant adeno-associated viral vector transduction

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12 December 2020

Recombinant adeno-associated viral vector encoding human VEGF165 induces neomicrovessel formation in the adult mouse brain.

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12 December 2020

Adeno-associated virus capsids displaying immunoglobulin-binding domains permit antibody-mediated vector retargeting to specific cell surface receptors

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12 December 2020

Correction of glycogen storage disease type II by an adeno-associated virus vector containing a muscle-specific promoter

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12 December 2020

Functional characterization of a recombinant adeno-associated virus 5-pseudotyped cystic fibrosis transmembrane conductance regulator vector

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12 December 2020

Subcellular compartmentalization of adeno-associated virus type 2 assembly

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12 December 2020

Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy

1 reference

12 December 2020

Differential myocardial gene delivery by recombinant serotype-specific adeno-associated viral vectors

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12 December 2020

Improving rAAV production and purification: towards the definition of a scaleable process

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12 December 2020

Adeno-associated viruses undergo substantial evolution in primates during natural infections

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12 December 2020

Adeno-associated viruses: an update.

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12 December 2020

Adeno-associated viral vectors for clinical gene transfer studies

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12 December 2020

Phase I trial of intranasal and endobronchial administration of a recombinant adeno-associated virus serotype 2 (rAAV2)-CFTR vector in adult cystic fibrosis patients: a two-part clinical study

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12 December 2020

Phase I clinical trial utilizing gene therapy for limb girdle muscular dystrophy: alpha-, beta-, gamma-, or delta-sarcoglycan gene delivered with intramuscular instillations of adeno-associated vectors

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12 December 2020

Human blue-opsin promoter preferentially targets reporter gene expression to rat s-cone photoreceptors

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12 December 2020

Recombinant adeno-associated virus serotype 9 leads to preferential cardiac transduction in vivo

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12 December 2020

Adeno-associated virus serotype 9 vectors transduce murine alveolar and nasal epithelia and can be readministered.

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12 December 2020

Efficient gene transfer of CD40 ligand into ovarian carcinoma cells with a recombinant adeno-associated virus vector.

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12 December 2020

Production, purification, crystallization and preliminary X-ray analysis of adeno-associated virus serotype 8.

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12 December 2020

Heparan sulfate proteoglycan binding properties of adeno-associated virus retargeting mutants and consequences for their in vivo tropism

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12 December 2020

Binding of adeno-associated virus type 5 to 2,3-linked sialic acid is required for gene transfer

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12 December 2020

Adeno-associated virus type 6 (AAV6) vectors mediate efficient transduction of airway epithelial cells in mouse lungs compared to that of AAV2 vectors

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12 December 2020

Circular intermediates of recombinant adeno-associated virus have defined structural characteristics responsible for long-term episomal persistence in muscle tissue

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12 December 2020

Infectious titer assay for adeno-associated virus vectors with sensitivity sufficient to detect single infectious events

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12 December 2020

Recombinant adeno-associated and adenoviral vectors for the transduction of pancreatic and colon carcinoma

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12 December 2020

Vascular bed-targeted in vivo gene delivery using tropism-modified adeno-associated viruses

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12 December 2020

Widespread and stable pancreatic gene transfer by adeno-associated virus vectors via different routes.

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12 December 2020

Impact of capsid conformation and Rep-capsid interactions on adeno-associated virus type 2 genome packaging

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12 December 2020

High levels of persistent expression of alpha1-antitrypsin mediated by the nonhuman primate serotype rh.10 adeno-associated virus despite preexisting immunity to common human adeno-associated viruses

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12 December 2020

AlphaVbeta5 integrin: a co-receptor for adeno-associated virus type 2 infection

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12 December 2020

Cell proliferation in the mammalian lung.

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12 December 2020

Identification of PDGFR as a receptor for AAV-5 transduction

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12 December 2020

Enhancing rAAV vector expression in the lung

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12 December 2020

Adeno-associated virus serotypes 1 to 5 mediated tumor cell directed gene transfer and improvement of transduction efficiency

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12 December 2020

Adeno-associated virus type 2 VP2 capsid protein is nonessential and can tolerate large peptide insertions at its N terminus

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12 December 2020

Successful gene transfer using adeno-associated virus vectors into the kidney: comparison among adeno-associated virus serotype 1-5 vectors in vitro and in vivo

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12 December 2020

Adeno-associated virus vectors can be efficiently produced without helper virus

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12 December 2020

Mutations in the carboxy terminus of adeno-associated virus 2 capsid proteins affect viral infectivity: lack of an RGD integrin-binding motif

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12 December 2020

Transfection-free and scalable recombinant AAV vector production using HSV/AAV hybrids

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12 December 2020

Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy

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12 December 2020

Maxillary sinusitis as a surrogate model for CF gene therapy clinical trials in patients with antrostomies

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12 December 2020

Comparison of adenoviral and adeno-associated viral vectors for pancreatic gene delivery in vivo

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12 December 2020

Cross-dressing the virion: the transcapsidation of adeno-associated virus serotypes functionally defines subgroups

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12 December 2020

Efficient AAV1-AAV2 hybrid vector for gene therapy of hemophilia

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12 December 2020

Structure determination of minute virus of mice

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12 December 2020

Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B.

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12 December 2020

A dose-ranging study of AAV-hAADC therapy in Parkinsonian monkeys

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12 December 2020

Molecular structure of adeno-associated virus variant DNA

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12 December 2020

Targeting recombinant adeno-associated virus vectors to enhance gene transfer to pancreatic islets and liver

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12 December 2020

Efficient gene delivery to human and rodent islets with double-stranded (ds) AAV-based vectors

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12 December 2020

Aspartoacylase gene transfer to the mammalian central nervous system with therapeutic implications for Canavan disease

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12 December 2020

Recombinant adeno-associated virus type 2, 4, and 5 vectors: transduction of variant cell types and regions in the mammalian central nervous system.

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12 December 2020

Adeno-associated virus vector serotypes mediate sustained correction of bilirubin UDP glucuronosyltransferase deficiency in rats

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12 December 2020

Enhanced transduction of mouse bone marrow-derived dendritic cells by repetitive infection with self-complementary adeno-associated virus 6 combined with immunostimulatory ligands

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12 December 2020

Sustained peripheral expression of transgene adiponectin offsets the development of diet-induced obesity in rats

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12 December 2020

Correction of the enzymatic and functional deficits in a model of Pompe disease using adeno-associated virus vectors

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12 December 2020

Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions

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12 December 2020

Overexpression of soluble TRAIL induces apoptosis in human lung adenocarcinoma and inhibits growth of tumor xenografts in nude mice.

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12 December 2020

AAV2-mediated gene delivery to monkey putamen: evaluation of an infusion device and delivery parameters

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12 December 2020

Alpha2,3 and alpha2,6 N-linked sialic acids facilitate efficient binding and transduction by adeno-associated virus types 1 and 6

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12 December 2020

Long-term correction of obesity and diabetes in genetically obese mice by a single intramuscular injection of recombinant adeno-associated virus encoding mouse leptin

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12 December 2020

Hepatocyte growth factor receptor is a coreceptor for adeno-associated virus type 2 infection.

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12 December 2020

Molecular characterization of adeno-associated viruses infecting children

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12 December 2020

Integration of adeno-associated virus (AAV) and recombinant AAV vectors

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12 December 2020

Several log increase in therapeutic transgene delivery by distinct adeno-associated viral serotype vectors

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12 December 2020

Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system

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12 December 2020

Mosaic vectors comprised of modified AAV1 capsid proteins for efficient vector purification and targeting to vascular endothelial cells

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12 December 2020

Immune responses to AAV in a phase I study for Canavan disease.

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12 December 2020

Development and optimization of a real-time quantitative PCR-based method for the titration of AAV-2 vector stocks

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12 December 2020

Identification and characterization of novel adeno-associated virus isolates in ATCC virus stocks

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12 December 2020

The atomic structure of adeno-associated virus (AAV-2), a vector for human gene therapy.

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12 December 2020

Gene therapy for hemophilia B: AAV-mediated transfer of the gene for coagulation factor IX to human muscle.

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12 December 2020

Purification of recombinant adeno-associated virus vectors by column chromatography and its performance in vivo

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12 December 2020

Anti-Abeta single-chain antibody delivery via adeno-associated virus for treatment of Alzheimer's disease

1 reference

12 December 2020

Oral adeno-associated virus-sTRAIL gene therapy suppresses human hepatocellular carcinoma growth in mice

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12 December 2020

Insertional mutagenesis at positions 520 and 584 of adeno-associated virus type 2 (AAV2) capsid gene and generation of AAV2 vectors with eliminated heparin- binding ability and introduced novel tropism

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12 December 2020

The VP1 capsid protein of adeno-associated virus type 2 is carrying a phospholipase A2 domain required for virus infectivity

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12 December 2020

Clinical protocol. Gene therapy of Canavan disease: AAV-2 vector for neurosurgical delivery of aspartoacylase gene (ASPA) to the human brain

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12 December 2020

Liver transduction with recombinant adeno-associated virus is primarily restricted by capsid serotype not vector genotype

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12 December 2020

Altering AAV tropism with mosaic viral capsids.

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12 December 2020

Induction of robust immune responses against human immunodeficiency virus is supported by the inherent tropism of adeno-associated virus type 5 for dendritic cells

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12 December 2020

Recombinant AAV-mediated gene delivery to the central nervous system.

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12 December 2020

Structure of adeno-associated virus type 4.

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12 December 2020

Characterization of adeno-associated virus genomes isolated from human tissues

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12 December 2020

Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy

1 reference

12 December 2020

A phase I study of aerosolized administration of tgAAVCF to cystic fibrosis subjects with mild lung disease

1 reference

12 December 2020

Highly purified recombinant adeno-associated virus vectors are biologically active and free of detectable helper and wild-type viruses

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12 December 2020

Human fibroblast growth factor receptor 1 is a co-receptor for infection by adeno-associated virus 2.

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12 December 2020

Adeno-associated virus serotype 4 (AAV4) and AAV5 both require sialic acid binding for hemagglutination and efficient transduction but differ in sialic acid linkage specificity

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12 December 2020

Mutational analysis of the adeno-associated virus type 2 (AAV2) capsid gene and construction of AAV2 vectors with altered tropism

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12 December 2020

Practical aspects of the development of ex vivo and in vivo gene therapy for Parkinson's disease

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12 December 2020

Adeno-associated virus-mediated gene transfer to hair cells and support cells of the murine cochlea

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12 December 2020

Assembly of adeno-associated virus

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12 December 2020

Persistent expression of canine factor IX in hemophilia B canines

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12 December 2020

Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1.

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12 December 2020

Convection-enhanced delivery of adeno-associated virus type 2 (AAV2) into the striatum and transport of AAV2 within monkey brain

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12 December 2020

Chronic recurrent myocardial ischemic injury is significantly attenuated by pre-emptive adeno-associated virus heme oxygenase-1 gene delivery

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12 December 2020

Transduction of dendritic cells by DNA viral vectors directs the immune response to transgene products in muscle fibers

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12 December 2020

Characterization of tissue tropism determinants of adeno-associated virus type 1.

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12 December 2020

AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B.

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12 December 2020

Intrathecal administration of AAV vectors for the treatment of lysosomal storage in the brains of MPS I mice

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12 December 2020

Recombinant adeno-associated virus (rAAV) expressing TFPI-2 inhibits invasion, angiogenesis and tumor growth in a human glioblastoma cell line.

1 reference

12 December 2020

High-titer recombinant adeno-associated virus production utilizing a recombinant herpes simplex virus type I vector expressing AAV-2 Rep and Cap.

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12 December 2020

Adeno-associated virus (AAV)-7 and -8 poorly transduce vascular endothelial cells and are sensitive to proteasomal degradation.

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12 December 2020

Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid

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12 December 2020

Enhancement of muscle gene delivery with pseudotyped adeno-associated virus type 5 correlates with myoblast differentiation.

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12 December 2020

Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors

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12 December 2020

Intranasal vaccination with recombinant adeno-associated virus type 5 against human papillomavirus type 16 L1

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12 December 2020

AAV8-mediated expression of glucocerebrosidase ameliorates the storage pathology in the visceral organs of a mouse model of Gaucher disease.

1 reference

12 December 2020

Genetic capsid modifications allow efficient re-targeting of adeno-associated virus type 2.

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12 December 2020

AAV serotype-1 mediates early onset of gene expression in mouse hearts and results in better therapeutic effect

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12 December 2020

Sustained correction of bleeding disorder in hemophilia B mice by gene therapy

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12 December 2020

Quantification of adeno-associated virus particles and empty capsids by optical density measurement

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12 December 2020

Capsid modifications overcome low heterogeneous expression of heparan sulfate proteoglycan that limits AAV2-mediated gene transfer and therapeutic efficacy in human ovarian carcinoma

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12 December 2020

Characteristics of a human cell line transformed by DNA from human adenovirus type 5

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12 December 2020

Synthesis of adeno-associated virus structural proteins requires both alternative mRNA splicing and alternative initiations from a single transcript.

1 reference

12 December 2020

Clinical protocol. Administration of a replication-deficient adeno-associated virus gene transfer vector expressing the human CLN2 cDNA to the brain of children with late infantile neuronal ceroid lipofuscinosis

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12 December 2020

Recombinant AAV-mediated HSVtk gene transfer with direct intratumoral injections and Tet-On regulation for implanted human breast cancer

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12 December 2020

Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart

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12 December 2020

Production methods for gene transfer vectors based on adeno-associated virus serotypes

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12 December 2020

Structure of adeno-associated virus serotype 5.

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12 December 2020

Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors

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12 December 2020

New recombinant serotypes of AAV vectors.

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12 December 2020

Insect cells as a factory to produce adeno-associated virus type 2 vectors

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12 December 2020

Long-term clinical improvement in MPTP-lesioned primates after gene therapy with AAV-hAADC.

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12 December 2020

Genetics of adeno-associated virus: isolation and preliminary characterization of adeno-associated virus type 2 mutants

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12 December 2020

Improved cardiac gene transfer by transcriptional and transductional targeting of adeno-associated viral vectors.

1 reference

12 December 2020

Down-regulation of alpha-synuclein expression can rescue dopaminergic cells from cell death in the substantia nigra of Parkinson's disease rat model

1 reference

12 December 2020

A shortened adeno-associated virus expression cassette for CFTR gene transfer to cystic fibrosis airway epithelia.

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12 December 2020

Adeno-associated virus (AAV) serotypes 2, 4 and 5 display similar transduction profiles and penetrate solid tumor tissue in models of human glioma

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12 December 2020

Subthalamic GAD gene transfer in Parkinson disease patients who are candidates for deep brain stimulation.

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12 December 2020

Mechanisms of adeno-associated virus genome encapsidation

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12 December 2020

High throughput production, screening and analysis of adeno-associated viral vectors

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12 December 2020

Monoclonal antibodies against the adeno-associated virus type 2 (AAV-2) capsid: epitope mapping and identification of capsid domains involved in AAV-2-cell interaction and neutralization of AAV-2 infection

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12 December 2020

Assembly of adeno-associated virus type 2 capsids in vitro.

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12 December 2020

Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors

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12 December 2020

Adeno-associated virus-mediated aspartoacylase gene transfer to the brain of knockout mouse for canavan disease

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12 December 2020

Structure, sequence, and function correlations among parvoviruses

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12 December 2020

Adeno-associated virus type 2 contains an integrin alpha5beta1 binding domain essential for viral cell entry

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12 December 2020

Adeno-associated virus 2-mediated intratumoral prostate cancer gene therapy: long-term maspin expression efficiently suppresses tumor growth

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12 December 2020

Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector.

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12 December 2020

Immune responses to adeno-associated virus vectors

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12 December 2020

In vivo expression of therapeutic human genes for dopamine production in the caudates of MPTP-treated monkeys using an AAV vector

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12 December 2020

Adeno-associated virus types 5 and 6 use distinct receptors for cell entry

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12 December 2020

Adeno-associated virus-mediated delivery of a mutant endostatin suppresses ovarian carcinoma growth in mice

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12 December 2020

Helper virus-free, optically controllable, and two-plasmid-based production of adeno-associated virus vectors of serotypes 1 to 6.

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12 December 2020

Therapeutic levels of factor IX expression using a muscle-specific promoter and adeno-associated virus serotype 1 vector

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12 December 2020

Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity

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12 December 2020

Canine parvovirus capsid structure, analyzed at 2.9 A resolution

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12 December 2020

Viral serotype and the transgene sequence influence overlapping adeno-associated viral (AAV) vector-mediated gene transfer in skeletal muscle

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12 December 2020

Repeat transduction in the mouse lung by using adeno-associated virus vectors with different serotypes

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12 December 2020

Hematopoietic stem cell transduction by recombinant adeno-associated virus vectors: problems and solutions

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12 December 2020

Recombinant AAV serotype 1 transduction efficiency and tropism in the murine brain

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12 December 2020

Successful production of pseudotyped rAAV vectors using a modified baculovirus expression system

1 reference

12 December 2020

Correction of Hunter syndrome in the MPSII mouse model by AAV2/8-mediated gene delivery

1 reference

12 December 2020

Muscle-directed gene transfer and transient immune suppression result in sustained partial correction of canine hemophilia B caused by a null mutation

1 reference

12 December 2020

Efficacy and safety of adeno-associated viral vectors based on serotype 8 and 9 vs. lentiviral vectors for hemophilia B gene therapy

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12 December 2020

Widespread and early myocardial gene expression by adeno-associated virus vector type 6 with a beta-actin hybrid promoter.

1 reference

12 December 2020

Safety and biological efficacy of an adeno-associated virus vector-cystic fibrosis transmembrane regulator (AAV-CFTR) in the cystic fibrosis maxillary sinus.

1 reference

12 December 2020

AAV-mediated leptin receptor installation improves energy balance and the reproductive status of obese female Koletsky rats

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12 December 2020

Attenuation of dengue virus infection by adeno-associated virus-mediated siRNA delivery

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12 December 2020

Determination of specific CD4 and CD8 T cell epitopes after AAV2- and AAV8-hF.IX gene therapy

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12 December 2020

Intracranial delivery of CLN2 reduces brain pathology in a mouse model of classical late infantile neuronal ceroid lipofuscinosis.

1 reference

12 December 2020

Adeno-associated virus vectors serotyped with AAV8 capsid are more efficient than AAV-1 or -2 serotypes for widespread gene delivery to the neonatal mouse brain

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12 December 2020

Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy

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12 December 2020

Adeno-associated virus 2-mediated transduction and erythroid lineage-restricted long-term expression of the human beta-globin gene in hematopoietic cells from homozygous beta-thalassemic mice

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12 December 2020

Evaluation of primitive murine hematopoietic stem and progenitor cell transduction in vitro and in vivo by recombinant adeno-associated virus vector serotypes 1 through 5.

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12 December 2020

Anti-angiogenic therapy subsequent to adeno-associated-virus-mediated immunotherapy eradicates lymphomas that disseminate to the liver

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12 December 2020

Sustained and complete phenotype correction of hemophilia B mice following intramuscular injection of AAV1 serotype vectors

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12 December 2020

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

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12 December 2020

One year transgene expression with adeno-associated virus cardiac gene transfer

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12 December 2020

Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver

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12 December 2020

Improved hepatic gene transfer by using an adeno-associated virus serotype 5 vector

1 reference

12 December 2020

DNA helicase-mediated packaging of adeno-associated virus type 2 genomes into preformed capsids

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12 December 2020

Development of a safe oral Abeta vaccine using recombinant adeno-associated virus vector for Alzheimer's disease.

1 reference

12 December 2020

Circulating anti-wild-type adeno-associated virus type 2 (AAV2) antibodies inhibit recombinant AAV2 (rAAV2)-mediated, but not rAAV5-mediated, gene transfer in the brain

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12 December 2020

Structurally mapping the diverse phenotype of adeno-associated virus serotype 4.

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12 December 2020

Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia

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12 December 2020

Electron cryo-microscopy and image reconstruction of adeno-associated virus type 2 empty capsids

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12 December 2020

Targeted gene delivery to vascular tissue in vivo by tropism-modified adeno-associated virus vectors

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12 December 2020

Development and characterization of a cell line for large-scale, serum-free production of recombinant adeno-associated viral vectors

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12 December 2020

Attachment of adeno-associated virus type 3H to fibroblast growth factor receptor 1.

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12 December 2020

10.1007/978-1-59745-210-6_2

Identifiers

DOI

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18 August 2017

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