Recombinant Adeno Associated Viral Vector
Zhang, Wenli (2012): Adenovirus-based gene therapy approaches for hemophilia B. Dissertation, LMU München: Medizinische Fakultät
... Another vector that holds great promise for hemophilia gene therapy is represented by recombinant adeno-associated viral vector ...an adeno-associated viral ...
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Intracellular Viral Processing, Not Single-Stranded DNA Accumulation, Is Crucial for Recombinant Adeno-Associated Virus Transduction
... after vector administration and require approximately 4 to 6 weeks before a plateau is ...AAV vector genomes revealed a variety of circular molecules con- verted from AAV single-stranded DNA genomes both in ...
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Characterization of Wild-Type Adeno-Associated Virus Type 2-Like Particles Generated during Recombinant Viral Vector Production and Strategies for Their Elimination
... third approach in which the replication-competent replicative DNA intermediates of wt AAV-like genomes were cloned into a plasmid and subjected to nucleotide sequence analyses. The use of restriction enzyme BalI ensured ...
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Rescue and replication of adeno-associated virus type 2 as well as vector DNA sequences from recombinant plasmids containing deletions in the viral inverted terminal repeats: selective encapsidation of viral genomes in progeny virions.
... for viral DNA replication. These include the viral rep gene, which codes for four nonstructural proteins that are synthesized from a single open reading frame by the use of alternate promoters and splicing ...
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Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA.
... CAAG-FIX vector that has a constitutive CMV enhancer-  -actin promoter combination as giving the highest levels among the 4 vectors tested regardless of whether the vector was administered by the ...
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Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques
... with vector plasmid and subjected to PCR to ensure that the sample did not contain inhibitors of ...the viral DNA was extracted and subjected to the PCR conditions described ...500 vector particles ...
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Isolation of Recombinant Adeno-Associated Virus Vector-Cellular DNA Junctions from Mouse Liver
... potential vector for gene therapy ...by recombinant AAV (rAAV) vectors (21, ...rAAV vector integration into the host ...only vector element necessary for integration (38), but the efficiency ...
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Incorporation of Antigens into Viral Capsids Augments Immunogenicity of Adeno-Associated Virus Vector-Based Vaccines
... group of mice (n ⫽ 6), which had been injected with 10 g of recombinant Ag85A protein subcutaneously, served as a positive control. Sera of BALB/c mice collected prior to injection (not shown) and 2 (A), 4 (B) ...
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Adeno-Associated Viral Vector-Driven Expression of Coagulation Proteins for Treatment of Hemophilias and Cancer
... Xigris, recombinant human activated protein C intended for use in sepsis treatment, from the market after failed efficacy and bleeding ...of recombinant human zyPC into septic, but otherwise healthy, ...
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Viral expression of ALS-linked ubiquilin-2 mutants causes inclusion pathology and behavioral deficits in mice
... into recombinant adeno-associated viral (rAAV) vectors, serotype 2, with expression cassette containing a cytomegalovirus enhancer/chicken beta actin (CBA) promoter, bovine growth hormone ...
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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.
... 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, ...Although recombinant AAV vec- tors ...
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Liver Transduction with Recombinant Adeno-Associated Virus Is Primarily Restricted by Capsid Serotype Not Vector Genotype
... the viral serotype, attempts to improve AAV gene transfer by varying the vector genotype have been rarely re- ported to ...the viral DNA (1, 3, 56, 61, 65, 66, 69) (see also ...the vector ...
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Kinetics of Recombinant Adeno-Associated Virus-Mediated Gene Transfer
... The viral genome contains two open reading frames that express four Rep and three Cap proteins and is flanked by the 145-bp terminal repeats, which are the only cis-acting elements that are essential for replica- ...
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Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway.
... incubated for 30 minutes at room temperature with the Cy3 dye in a reaction volume of 1 mL. The solutions were then transferred to dialysis cassettes (10 000 molecular weight cutoff; Pierce Biotechnology, Rockford, IL) ...
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Disease-Inducible Transgene Expression from a Recombinant Adeno-Associated Virus Vector in a Rat Arthritis Model
... rAAV. The rAAV-lacZ construct used in this work contains a lacZ reporter gene that harbors a nuclear localization signal under the regulation of the CMV immediate-early promoter (42). Preparations of the rAAV-lacZ ...
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Circumventing furin enhances factor VIII biological activity and ameliorates bleeding phenotypes in hemophilia models
... or recombinant FVIII ...of adeno-associated viral (AAV) vector liver-directed gene therapy of factor IX (FIX) for hemophilia B (HB) engenders optimism that FVIII gene therapeutics may ...
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Genetic Fate of Recombinant Adeno-Associated Virus Vector Genomes in Muscle
... rAAV vector DNA was persist- ing as extrachromosomal high-molecular-weight ...the viral ITR, an observation consistent with previous in vivo and in vitro data showing significant rearrangement of ITR ...
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Recombinant Adeno-Associated Viral Vectors Are Deficient in Provoking a DNA Damage Response
... foci associated with AAV2 DNA. Here, we investigated whether recombinant AAV2 (rAAV2) vectors are able to produce a similar ...or viral capsid ...rAAV2 vector containing the p5 AAV2 sequence ...
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rAAV mediated overexpression of TGF β stably restructures human osteoarthritic articular cartilage in situ
... of recombinant factor were applied (100–200 ng while we report up to ...5 viral particles) ...gene vector and recombinant factor can target all the tissues of the joint, allowing TGF-β to ...
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Extrachromosomal Recombinant Adeno-Associated Virus Vector Genomes Are Primarily Responsible for Stable Liver Transduction In Vivo
... kb. Recombinant AAV (rAAV) vectors based on AAV type 2 are of great interest in the field of gene therapy for monogenic diseases because they can safely direct persistent transgene expression from transduced ...
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