METHOD FOR GENERATING HIGH TITER HELPER-FREE PREPARATION OF RECOMBINANT AAV VECTOR

PROBLEM TO BE SOLVED: To provide a method and a composition for producing high titer, substantially purified preparation of a recombinant adeno-associated virus (AAV) that can be used as a vector for gene delivery. SOLUTION: At the onset of vector production, the AAV producer cells typically compris...

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Hauptverfasser: REYNOLDS THOMAS C, FUNG VICTOR P, ATKINSON EDWARD MORROW, WILKINS PERRY C, TAKEYA RYAN K
Format: Patent
Sprache:eng
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Zusammenfassung:PROBLEM TO BE SOLVED: To provide a method and a composition for producing high titer, substantially purified preparation of a recombinant adeno-associated virus (AAV) that can be used as a vector for gene delivery. SOLUTION: At the onset of vector production, the AAV producer cells typically comprise one or more AAV packaging genes, an AAV vector comprising a heterologous (i.e. non-AAV) transgene of interest, and a helper virus such as an adenovirus. The AAV vector preparation produced can mediate delivery of a transgene of interest (e.g. a therapeutic gene) to any of a wide variety of tissues and cells. The AAV vector preparation is also substantially free of helper virus as well as helper viral and cellular proteins and other contaminants. Also there is provided a quantitative, high-throughput assay useful in the assessment of the screening of an agent that affects viral infectivity and/or replication. COPYRIGHT: (C)2010,JPO&INPIT