The adeno-associated viral vector system
The Impact of Peptide Insertions on Adeno-Associated Viral Vector Fate
... the vector dose in case of the HSPG- non-binder mutants A2 and ...Second, vector uncoating could have been impaired in case of proteoglycan- dependently internalized vectors, and third, vector ...
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Adeno-Associated Viral Vectors
... A description of how the hazardous materials are to be used in the protocol. See the following example: “The protocol will be conducted at Biosafety Level 2 (BSL2) and Animal Biosafety Level 1 (ABSL1). Staff will use ...
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Vector uncoating limits adeno-associated viral vector-mediated transduction of human dendritic cells and vector immunogenicity
... associated viral vector-mediated transduction of human dendritic cells and vector immunogenicity Axel Rossi 1,2 , Léa Dupaty 3 , Ludovic Aillot 1,8 , Liang Zhang 4 , Célia Gallien 1 , Michael ...
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Adeno-Associated Viral Vector-Driven Expression of Coagulation Proteins for Treatment of Hemophilias and Cancer
... efficacious vector doses in order to prevent anti-AAV capsid CD8 + cytotoxic T cells from destroying the transduced hepatocytes and resulting in loss of transgene ...that vector doses for HB can be ...
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Incorporation of Antigens into Viral Capsids Augments Immunogenicity of Adeno-Associated Virus Vector-Based Vaccines
... FIG 1 Western blot of AAV preparations. Four different AAV vectors (Ag85A-AAV:Ag85A, Ag85A-AAV:GFP, AAV:Ag85A, and AAV:GFP) were assayed for induction of humoral immune responses. Equal genomic particles of each ...
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Adeno-associated virus vector integration junctions.
... single vector provirus copy (data not ...AAV-SNori vector genome including both TRs and depends on the p15A replication origin in the vector sequences for replication, making it similar in structure ...
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Adeno-Associated Virus as a Vector for Liver-Directed Gene Therapy
... each vector was essen- tially the same, with the concentration of serum a-1AT in- creasing during the first 6 weeks and thereafter stabilizing for the duration of the ...of vector and transgene expression ...
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In vivo model of adeno-associated virus vector persistence and rescue.
... The rescue of vector DNA sequences may also be relevant to the risk of shedding of AAV-CFTR after delivery to the airway surface. The lack of detectable integration in this model is interest- ing. In latent ...
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Recent advances in recombinant adeno-associated virus vector production
... rAAV vector produc- simplified method for generating rAAV based on stable tion was the development of helper virus-free packaging HeLa cell lines containing integrated copies of the AAV systems that use a DNA ...
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Adeno associated virus: from defective virus to effective vector
... the viral encoding regions, rAAV design can follow the whole- gene-removal or "gutless" vector rational of, for instance, retrovirus-based vectors in the sense that the cis-acting ele- ments involved in ...
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Influence of the SUMOylation pathway on adeno-associated virus vector transduction
... 11 Figure 4 Schematic representation of Adeno-Associated Virus life cycle. AAV2 binds onto the surface of a target cell via receptor and/or co-receptor facilitates the internalization of the virus. Before ...
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Creation of a cardiotropic adeno associated virus: the story of viral directed evolution
... One classic method to modify the tropism of a viral vector was to insert a ligand on the virus surface to tar- get the specific cell type. Initially, the targeting ligand was isolated via an indirect ...
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Improving safety and establishing episomal maintenance of Adeno-associated viral vectors
... AAV vector transduction is increased in Ku80-defective ...AAV vector transduction. 177 Furthermore, AAV vector transduction was increased in vitro upon administration of drugs which induce transient ...
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Structure of Adeno-Associated Virus Serotype 8, a Gene Therapy Vector
... Summary. The 2.6-Å-resolution structure presented for AAV8 further highlights the high overall homology between the VP structures of the AAVs, which likely dictates successful assembly. Structural differences between ...
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Genetic Fate of Recombinant Adeno-Associated Virus Vector Genomes in Muscle
... rAAV vector DNA hybridizing species, in- cluding high-molecular-weight forms, a monomeric vector form, and residual input single-stranded vector ...rAAV vector, the high-molecular-weight and ...
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Intrathecal Adeno-Associated Virus Vector Delivery for Mucopolysaccharidosis Type I
... However, of the three animals treated with intrathecal vector alone, two developed sufficiently robust anti-transgene antibody responses to prevent complete resolution of CNS storage lesions. Only the animal that ...
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A Single Vector Platform for High-Level Gene Transduction of Central Neurons: Adeno-Associated Virus Vector Equipped with the Tet-Off System.
... AAV vector containing the CMV promoter, the difference in GFP-NF intensity in Neuro-2a cells between the two vectors was only ...AAV-SynTetOff vector may be different in various cell types, and this needs ...
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Recombinant Adeno-Associated Viral Vectors Are Deficient in Provoking a DNA Damage Response
... Adeno-associated virus type 2 (AAV2) provokes a DNA damage response that mimics a stalled replication fork. We have previously shown that this response is dependent on ataxia telangiectasia-mutated and ...
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Host Cell DNA Repair Pathways in Adeno-Associated Viral Genome Processing
... of cells was infected when scAAVGFP-cd reagents were tested. In addition, in order to measure only expression due to circularization of monomeric genomes and not from concate- merized genomes, we used a multiplicity of ...
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Adeno-Associated Virus Vector Genomes Persist as Episomal Chromatin in Primate Muscle
... rAAV vector genomes exist as episomal circles in NHP mus- ...a vector-specific WPRE-pA ...resistant vector-specific bands that represent circular super- coiled and relaxed monomers and circular HMW ...
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