Abstract
Recombinant adeno-associated viral (rAAV) vectors are being evaluated in animal models and humans. Pre-clinical data demonstrating vector safety, efficiency and efficacy have been used to initiate human clinical trials. The clinical manufacture of rAAV vectors has supported phase I and phase II trials, showing that adeno-associated virus serotype 2 vectors are safe when administered to humans.
Publication types
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Research Support, Non-U.S. Gov't
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Research Support, U.S. Gov't, P.H.S.
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Review
MeSH terms
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Animals
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Clinical Trials as Topic
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Cystic Fibrosis / therapy
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DNA, Recombinant / genetics
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DNA, Recombinant / therapeutic use*
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Dependovirus / genetics*
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Dependovirus / isolation & purification
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Gene Expression Regulation, Viral
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Gene Transfer Techniques
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Genetic Therapy / methods*
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Genetic Vectors / genetics*
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Genetic Vectors / isolation & purification
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Genetic Vectors / therapeutic use*
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Genome, Viral
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Humans
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Recombination, Genetic*