adeno-associated virus vector
Adeno-associated virus vector integration junctions.
... DNA techniques. Restriction enzymes, T4 DNA ligase, and DNA polymerases were from New England BioLabs, Beverly, Mass. Proteinase K was from Boehr- inger Mannheim. Enzyme reactions were performed by using the ...
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Widespread Gene Delivery and Structure-Specific Patterns of Expression in the Brain after Intraventricular Injections of Neonatal Mice with an Adeno-Associated Virus Vector
... In the olfactory bulb, the principal neurons are mitral cells, which send long axonal projections via the lateral olfactory tract to the paleocortex. An extensive pattern of transduction was observed in the mitral cell ...
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Adeno-Associated Virus Vector Genomes Persist as Episomal Chromatin in Primate Muscle
... the vector DNA is concomitant with the rise of transgene expression ...different vector copy numbers, and (viii) different species (mice, NHPs) (references 44, 64, 65, and 66 and data presented ...
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Potent inhibition of human immunodeficiency virus type 1 in primary T cells and alveolar macrophages by a combination anti-Rev strategy delivered in an adeno-associated virus vector.
... It is also noteworthy that, in this strategy, both a protein and a nucleic acid were employed together to target two distinct domains in a single key viral protein. The RRE binding domain targeted by the decoy is ...
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Genetic Fate of Recombinant Adeno-Associated Virus Vector Genomes in Muscle
... Recombinant adeno-associated virus (rAAV) vectors are promising human gene transfer vectors, because they mediate long-term gene expression in ...The vector DNA form responsible for sustained ...
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Novel Adeno-Associated Virus Vector Vaccine Restricts Replication of Simian Immunodeficiency Virus in Macaques
... the vector genome are the noncoding ITRs (145 ...rAAV vector vaccines might benefit from a chemical or molecular adjuvant like those used to improve plasmid DNA vaccines (5–7, 23, 41, 73, ...
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Large-Scale Molecular Characterization of Adeno-Associated Virus Vector Integration in Mouse Liver
... compared vector-flanking DNA sequences against human genome DNA using the same programs described above or against DNA databases of all ...the vector-flanking DNA sequence with the se- quence of the ...
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Structure of Adeno-Associated Virus Vector DNA following Transduction of the Skeletal Muscle
... the vector ssDNA into a ds form that can serve as a transcription tem- plate (11, ...and vector genome status in the permissive context of the skeletal ...this vector DNA comes from particles trapped ...
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Intrathecal Adeno-Associated Virus Vector Delivery for Mucopolysaccharidosis Type I
... the adeno- associated virus genome could be provided in trans, allowing for the generation of infectious particles in which the endogenous genome was replaced with a transgene (Samulski, Chang, and ...
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In vivo model of adeno-associated virus vector persistence and rescue.
... and adeno-associated virus (AAV) vectors but has not previously been studied in ...AAV vector (AAV-CFTR) with wt AAV type 2 (AAV2) and a host range mutant Ad (Ad2HR405) for which monkey cells ...
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Developments in the treatment of hemophilia B: focus on emerging gene therapy
... viral vector systems each have unique properties that influence their ability to deliver genes to different tissues, and the data generated in several clinical trials testing different vectors for hemophilia have ...
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Adeno-Associated Virus as a Vector for Liver-Directed Gene Therapy
... spleen. Vector constructs contained the EcoRI fragment of pAT85 (from the American Type Culture Collection), spanning human a-1AT cDNA fused to the polyadenylation sequence from simian virus ...leukemia ...
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A cis-Acting Element That Directs Circular Adeno-Associated Virus Replication and Packaging
... Construction of mutant cAAV vectors. The structures of recombinant cAAV genomes are schematically presented in Fig. 1. All plasmids of this set harbor an enhanced green fluorescent protein (EGFP) under the control of the ...
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Adeno associated virus: from defective virus to effective vector
... helper virus such as Ad or her- pes simplex virus (HSV) whereas the latter is established in host cells in the absence of a helper ...the virus genome into a region of roughly 2-kb on the long arm ...
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Vector design influences hepatic genotoxicity after adeno associated virus gene therapy
... a vector has been challenged by a single study that documented hepatocellular carcinoma (HCC) after AAV gene delivery in ...were associated with HCC. In addition, we demonstrated that the AAV vector ...
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Adeno-Associated Virus Production in Serum-Free Suspension Adapted HEK293 Cells for Gene Therapy.
... simplex virus type-1 (HSV 1) are used as gene therapy vectors and can be prepared in one of two ...HSV virus, which results in amplicon packaged viral ...plasmid vector viral sequences and the ...
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Residues on Adeno-associated Virus Capsid Lumen Dictate Interactions and Compatibility with the Assembly-Activating Protein
... AAP must interact with VP in a specific way to execute its numerous functions, but the nature of the specific AAP-VP interaction is not well understood. The AAPs of most serotypes are compatible with VPs of heterologous ...
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Adeno-Associated Virus Type 2 Wild-Type and Vector-Mediated Genomic Integration Profiles of Human Diploid Fibroblasts Analyzed by Third-Generation PacBio DNA Sequencing
... amplify virus- chromosome junctions a linker-based strategy was applied as described previously ...fragments. Virus-chromosome junctions derived from six restriction enzyme digests were amplified from ...
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Adeno-associated virus type 2 DNA replication in vivo: mutation analyses of the D sequence in viral inverted terminal repeats.
... Implications of the D sequence mutations in the develop- ment of AAV vectors for gene therapy. AAV has gained atten- tion as a potentially useful vector for human gene therapy, primarily because of its ...
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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.
... sarcoma virus long terminal repeat (RSV- LTR) promoter from plasmid cN61, were subcloned in be- tween the AAV ITRs derived from plasmid psub201 (33), to construct a recombinant AAV plasmid, designated pKY-1, by ...
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