C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 7/00 (2006.01) A61K 48/00 (2006.01) C12N 7/01 (2006.01) C12N 7/02 (2006.01) C12N 15/861 (2006.01)
Patent
CA 2682108
This invention provides methods and compositions for producing high titer, substantially purified preparations of recombinant adeno-associated virus (AAV) that can be used as vectors for gene delivery. At the onset of vector production, AAV producer cells of this invention typically comprise one or more AAV packaging genes, an AAV vector comprising a heterelogous (i.e. non-AAV) transgene of interest, and a helper virus such as an adenovirus. The AAV vector preparations produced are generally replication incompetent but are capable of mediating delivery of a transgene of interest (such as a therapeutic gene) to any of a wide variety of tissues and cells. The AAV vector preparations produced according to this invention are also substantially free of helper virus as well as helper viral and cellular proteins and other contaminants. Also provided is a quantitative, high-throughput assay useful in the assessment of viral infectivity and replication, as well as in the screening of agents that affect viral infectivity and/or replication.
Atkinson Edward Morrow
Fung Victor P.
Reynolds Thomas C.
Takeya Ryan K.
Wilkins Perry C.
Borden Ladner Gervais Llp
Genzyme Corporation
Targeted Genetics Corporation
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