[PDF] Top 20 Improved Hepatic Gene Transfer by Using an Adeno-Associated Virus Serotype 5 Vector
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Improved Hepatic Gene Transfer by Using an Adeno-Associated Virus Serotype 5 Vector
... these vector doses has not been demonstrated before using an AAV vector, and recent data from others have also demonstrated improved efficacy of hepatic gene transfer with ... See full document
6
Frequency and Spectrum of Genomic Integration of Recombinant Adeno-Associated Virus Serotype 8 Vector in Neonatal Mouse Liver
... recombinant adeno-associated virus serotype 8 (rAAV8) vectors results in widespread transduction in multiple organs and therefore holds promise in neonatal gene ...11 vector ... See full document
12
Adeno-Associated Virus Type 5 (AAV5) but Not AAV2 Binds to the Apical Surfaces of Airway Epithelia and Facilitates Gene Transfer
... the virus is applied to the basolateral surface ...markedly improved (1, 8). Although gene transfer may also be limited by other steps in the transduction process, such as inefficient ... See full document
7
Adeno associated virus: from defective virus to effective vector
... for gene transfer ...of gene delivery systems including those with a small packaging ...to 5, and > 5% correspond to severe, moderate and mild disease, ... See full document
17
Hybrid Vectors Based on Adeno-Associated Virus Serotypes 2 and 5 for Muscle-Directed Gene Transfer
... parvovirus adeno-associated virus (AAV) are being evaluated in preclinical and clinical models of gene ...on serotype 2 ...AAV2 vector transduction exists, ranging from skeletal ... See full document
5
Liver Transduction with Recombinant Adeno-Associated Virus Is Primarily Restricted by Capsid Serotype Not Vector Genotype
... with hepatic locus control region; bpA, bovine growth hormone poly(A) ...of vector genome copy ...after vector administration, and 10 g of DNA was analyzed by Southern blotting with BamHI/XhoI ... See full document
14
Endosomal processing limits gene transfer to polarized airway epithelia by adeno associated virus
... cells, 5 µL rAAV was mixed with 50 µL of culture media and applied directly onto the apical compartment of Millicell inserts (Millipore ...media. Gene transfer to the basal side was performed by ... See full document
16
Effect of Nuclear Factor κB Inhibition on Serotype 9 Adeno-Associated Viral (AAV9) Minidystrophin Gene Transfer to the mdx Mouse
... analyzed using primers and dual-hybridization labeled probes for (a) the human minidystrophin construct in the AAV9 minidystrophin vector and (b) murine GAPDH, which is a reference gene that permits ... See full document
11
Mapping a Neutralizing Epitope onto the Capsid of Adeno-Associated Virus Serotype 8
... he adeno-associated viruses (AAVs), members of the Depen- dovirus genus of the single-stranded DNA (ssDNA) Parvoviri- dae, are promising gene transfer vectors owing to their ability to ... See full document
13
Unrestricted Hepatocyte Transduction with Adeno-Associated Virus Serotype 8 Vectors in Mice
... liver gene therapy, but gene delivery to neurons and glial cells by viral vectors holds great promise for gene therapy for central nervous system ...utero gene transfer (26, 27) and ... See full document
11
High-Resolution Structural Characterization of a New Adeno-associated Virus Serotype 5 Antibody Epitope toward Engineering Antibody-Resistant Recombinant Gene Delivery Vectors
... defective gene for the treatment of monogenic ...in gene therapy is preexisting neutral- izing antibodies (NAbs) in a large percentage of the human population (16, ...AAV serotype (16, ...to ... See full document
16
Characterization of Fabry mice treated with recombinant adeno-associated virus 2/8-mediated gene transfer
... 2/8 vector, which purportedly produces more efficient hepatic transduction [14,21], would produce higher levels of α-Gal A expression and consequently greater affects on the pathology in the affected ... See full document
10
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 ... See full document
10
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 ... See full document
161
Enhancement of Muscle Gene Delivery with Pseudotyped Adeno-Associated Virus Type 5 Correlates with Myoblast Differentiation
... enhancing gene conversion ...of gene transfer with current AAV ...4, 5, 19, 23, ...a gene therapy ...in gene therapy applications. For example, AAV-5 likely utilizes a ... See full document
10
Structurally Mapping the Diverse Phenotype of Adeno-Associated Virus Serotype 4
... generated using the AAV2 inverted terminal repeats to package a re- porter gene (such as green fluorescent protein or luciferase) or a therapeutic gene within the capsids of other serotypes, have ... See full document
15
Kinetics of Recombinant Adeno-Associated Virus-Mediated Gene Transfer
... rAAV vector. The HMW DNA was subjected to Southern blot analysis using a CMV promoter- specific probe ...the 5⬘ end of the promoter and within the hFIX intron to release a ...(Fig. 5, lanes 10 ... See full document
11
Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector.
... rAAV-LacZ vector as episome, single integrated copy (monomer), or tandem (head-to-tail) and expected fragment sizes generated after EcoRV di- ...the vector fragment radiolabeled for Southern ...from ... See full document
11
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 ...early ... See full document
5
Structural Insights into Adeno-Associated Virus Serotype 5
... best-characterized serotype with respect to functional re- gions of the AAV VP or capsid is AAV2, facilitated by the available crystal structure of this serotype ...mostly associated with regions in ... See full document
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