Abstract
Adeno-associated virus (AAV)-based vectors expressing therapeutic gene products have shown great promise for human gene therapy. A major challenge for translation of promising research to clinical development is the establishment of appropriate quality control (QC) test methods to characterize clinical grade AAV vectors. This chapter focuses on QC testing, providing an overview of characterization methods appropriate for clinical vectors prepared for early phase clinical studies, and detailed descriptions for selected assays that are useful to assess AAV vector safety, potency, and purity.
Publication types
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Research Support, N.I.H., Extramural
MeSH terms
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Cell Culture Techniques / standards
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Cell Line
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Clinical Trials as Topic
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DNA, Recombinant / isolation & purification*
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Dependovirus / genetics*
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Dependovirus / isolation & purification
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Electrophoresis, Polyacrylamide Gel / methods
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Genetic Therapy / methods*
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Genetic Therapy / standards
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Genetic Vectors / isolation & purification*
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Genetic Vectors / standards
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Genome, Viral
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Guidelines as Topic
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Humans
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Polymerase Chain Reaction / standards
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Reference Standards
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Safety / standards*
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Titrimetry / methods
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Transduction, Genetic / methods
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Virion / genetics*
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Virion / isolation & purification
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Workflow