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Recombinant adeno-associated virus vector

Genetic Fate of Recombinant Adeno-Associated Virus Vector Genomes in Muscle

Genetic Fate of Recombinant Adeno-Associated Virus Vector Genomes in Muscle

... rAAV vector copy number in the muscle samples, 25 ng of total muscle DNA was used in a standard TaqMan PCR with the HCMV primer-probe ...resulting vector copy number was divided by 4,167 nuclei (assuming 6 ...

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Isolation of Recombinant Adeno-Associated Virus Vector-Cellular DNA Junctions from Mouse Liver

Isolation of Recombinant Adeno-Associated Virus Vector-Cellular DNA Junctions from Mouse Liver

... the vector plasmid molecules into 20 mg of total DNA extracted from naive mouse ...rAAV vector forms, because the same pattern was observed from the DNA extracted from rAAV stocks, in which only a ...

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C-Reactive Protein (CRP) Is Essential for Efficient Systemic Transduction of Recombinant Adeno-Associated Virus Vector 1 (rAAV-1) and rAAV-6 in Mice

C-Reactive Protein (CRP) Is Essential for Efficient Systemic Transduction of Recombinant Adeno-Associated Virus Vector 1 (rAAV-1) and rAAV-6 in Mice

... blood vector clearance, blood samples (50 ␮ l) were collected from the tail vein or retro- orbital plexus at 3, 6, 24, and 48 h postinjection using heparin-coated capillary ...

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Phenotypic correction of Fanconi anemia in human hematopoietic cells with a recombinant adeno associated virus vector

Phenotypic correction of Fanconi anemia in human hematopoietic cells with a recombinant adeno associated virus vector

... We demonstrated efficient transduction, expression, and phenotypic correction in lymphoblastoid cell lines derived from FA C patients using a recombinant adeno-associated virus rAAV vect[r] ...

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Disease-Inducible Transgene Expression from a Recombinant Adeno-Associated Virus Vector in a Rat Arthritis Model

Disease-Inducible Transgene Expression from a Recombinant Adeno-Associated Virus Vector in a Rat Arthritis Model

... a recombinant adeno-associated virus (rAAV) vector for its potential application in RA gene ...between vector transgene expression and disease severity in arthritic ...

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Extrachromosomal Recombinant Adeno-Associated Virus Vector Genomes Are Primarily Responsible for Stable Liver Transduction In Vivo

Extrachromosomal Recombinant Adeno-Associated Virus Vector Genomes Are Primarily Responsible for Stable Liver Transduction In Vivo

... We have previously suggested that the majority of rAAV genomes integrate as head-to-tail concatemers into chromo- somes in mouse hepatocytes in vivo after portal vein injection of vector (23). This was based on ...

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Liver Transduction with Recombinant Adeno-Associated Virus Is Primarily Restricted by Capsid Serotype Not Vector Genotype

Liver Transduction with Recombinant Adeno-Associated Virus Is Primarily Restricted by Capsid Serotype Not Vector Genotype

... AAV-5-genotyped vector DNA ...AAV vector geno- and ...AAV vector plasmid carrying the gfp gene flanked by ITRs from genotypes 1 through 6, an AAV helper plasmid expressing rep and cap genes from ...

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Frequency and Spectrum of Genomic Integration of Recombinant Adeno-Associated Virus Serotype 8 Vector in Neonatal Mouse Liver

Frequency and Spectrum of Genomic Integration of Recombinant Adeno-Associated Virus Serotype 8 Vector in Neonatal Mouse Liver

... rAAV vector integration site plasmid libraries and sequencing of plasmid ...rAAV vector integration site plasmid libraries were con- structed using ...AAV8-ISceI.AO3 vector-transduced mouse liver ...

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Novel Strategy for Generation and Titration of Recombinant Adeno-Associated Virus Vectors

Novel Strategy for Generation and Titration of Recombinant Adeno-Associated Virus Vectors

... helper virus for AAV replication ...animal virus as a helper virus to produce rAAV ...helper virus to provide replication and packaging functions for rAAV vec- tor ...been associated ...

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Kinetics of Recombinant Adeno-Associated Virus-Mediated Gene Transfer

Kinetics of Recombinant Adeno-Associated Virus-Mediated Gene Transfer

... rAAV vector genome. rAAV, delivered in the absence of helper virus and Rep pro- tein, persists as a single-stranded genome for a certain period in transduced ...

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Immunological Aspects of Recombinant Adeno-Associated Virus Delivery to the Mammalian Brain

Immunological Aspects of Recombinant Adeno-Associated Virus Delivery to the Mammalian Brain

... Recombinant adeno-associated viruses (rAAV) are highly efficient vectors for gene delivery into the central nervous system ...the vector and/or the transgene protein after repeated injections ...

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Adeno-Associated Virus as a Vector for Liver-Directed Gene Therapy

Adeno-Associated Virus as a Vector for Liver-Directed Gene Therapy

... a vector for in vivo gene therapy of the ...leukemia virus, and (v) the chicken b-actin promoter fused to the enhancer region of the CMV immediate early gene ...(CB). Recombinant stocks of AAV for ...

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Dynamin Is Required for Recombinant Adeno-Associated Virus Type 2 Infection

Dynamin Is Required for Recombinant Adeno-Associated Virus Type 2 Infection

... nonpathogenic virus capable of trans- ducing a broad range of cell types both in vitro and in vivo, this vector system has attracted tremendous interest in the field of gene ...this virus remains ...

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Generation of Neutralizing Activity against Human Immunodeficiency Virus Type 1 in Serum by Antibody Gene Transfer

Generation of Neutralizing Activity against Human Immunodeficiency Virus Type 1 in Serum by Antibody Gene Transfer

... immunodeficiency virus (HIV) vaccine approaches have elicited meaningful anti- gen-specific T-cell responses in animal models, no single vaccine candidate has engendered antibodies that broadly neutralize primary ...

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Adeno-associated virus type 2-mediated transfer of ecotropic retrovirus receptor cDNA allows ecotropic retroviral transduction of established and primary human cells.

Adeno-associated virus type 2-mediated transfer of ecotropic retrovirus receptor cDNA allows ecotropic retroviral transduction of established and primary human cells.

... The adeno-associated virus type 2 (AAV)-based vector sys- tem appears to be promising in overcoming some of the limi- tations of retrovirus vectors (4, 8, ...helper virus, the wild-type ...

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A cis-Acting Element That Directs Circular Adeno-Associated Virus Replication and Packaging

A cis-Acting Element That Directs Circular Adeno-Associated Virus Replication and Packaging

... of recombinant cAAV genomes are schematically presented in ...linear vector was derived from psub201 and contains a 13-bp deletion in the A region ...

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In vivo model of adeno-associated virus vector persistence and rescue.

In vivo model of adeno-associated virus vector persistence and rescue.

... the recombinant tgAAVCFTR vector ...that vector was detectable in the nose earlier than in the lung, given the site of the original ...that vector latency had occurred at both ...of ...

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Adeno-Associated Virus 8-Mediated Gene Therapy for Choroideremia: Preclinical Studies in in Vitro ind in Vivo Models

Adeno-Associated Virus 8-Mediated Gene Therapy for Choroideremia: Preclinical Studies in in Vitro ind in Vivo Models

... encoding recombinant adeno-associated virus serotype 8 (rAAV8) vector, which targets retinal cells efficiently, for therapeutic effect and safety in vitro and in vivo in a murine model ...

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A "humanized" green fluorescent protein cDNA adapted for high-level expression in mammalian cells.

A "humanized" green fluorescent protein cDNA adapted for high-level expression in mammalian cells.

... Construction of rAAV vector plasmids. Briefly, the gfp10 sequence was sub- cloned into the NotI site of pCMVb (Clontech) after digestion of the parent plasmid TU#65 (4) with AgeI and EcoRI, filling in the ends ...

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Feasibility of Generating Adeno-Associated Virus Packaging Cell Lines Containing Inducible Adenovirus Helper Genes

Feasibility of Generating Adeno-Associated Virus Packaging Cell Lines Containing Inducible Adenovirus Helper Genes

... The ultimate goal of our studies is to test the feasibility of creating ideal helper-virus-free AAV producer cell lines. The final AAV producer cell lines not only should harbor inte- grated and rescuable ...

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