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E. Ayuso et al.
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Luk H. Vandenberghe et al.
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Evidence for encapsidation of prokaryotic sequences during recombinant adeno-associated virus production and their in vivo persistence after vector delivery
G Chadeuf et al.
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AM Davidoff et al.
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Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors
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AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B
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Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivo
Z Wang et al.
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Quantification of adeno-associated virus particles and empty capsids by optical density measurement
JM Sommer et al.
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A versatile and scalable two-step ion-exchange chromatography process for the purification of recombinant adeno-associated virus serotypes-2 and-5
N Brument et al.
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Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy
GP Gao et al.
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AC Nathwani et al.
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Scalable purification of adeno-associated virus type 2, 4, or 5 using ion-exchange chromatography
N Kaludov et al.
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Gene therapy of Canavan disease:: AAV-2 vector for neurosurgical delivery of aspartoacylase gene (ASPA) to the human brain
C Janson et al.
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Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity
JE Rabinowitz et al.
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Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo
H Nakai et al.
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Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis
DM McCarty et al.
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Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA
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RR Ali et al.
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Endosomal processing limits gene transfer to polarized airway epithelia by adeno-associated virus
DS Duan et al.
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Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver
LL Wang et al.
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Repeat transduction in the mouse lung by using adeno-associated virus vectors with different serotypes
CL Halbert et al.
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