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AssigneesCell Genesys, Inc., The University of North Carolina at Chapel Hill
InventorsRichard Jude Samulski, Xiao Xiao, Richard Snyder
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The present invention relates to methods and compositions for increasing the production of high titre stocks of recombinant AAV (rAAV) through regulation of expression of the AAV REP and CAP proteins. The methods and compositions of the invention are based on the observation that the low level expression of the AAV REP protein increases the production of AAV viral capsid protein and efficiency of packaging resulting in production of higher titre recombinant viral stocks. The invention encompasses recombinant AAV vectors that direct the expression of AAV REP and CAP proteins and the use of such vectors for the production of novel stable cell lines capable of generating high titre rAAV vectors. The invention provides methods for regulating the expression of the AAV REP gene at the transcriptional and post-translational level. The methods and compositions of the invention can be used to produce high titre stocks of rAAV which can be used in gene therapy for the purpose of transferring genetic information into appropriate host cells for the management and correction of human diseases including inherited and acquired disorders.
A method for producing a stock of recombinant adeno-associated virus (AAV) comprising:
A host cell for producing a stock of recombinant adeno-associated virus containing a recombinant DNA vector wherein an AAV REP 78 or REP 68 protein is expressed under regulatory control of an AAV promoter, and wherein a decreased level of REP 78 or REP 68 protein is expressed relative to the level of REP 78 or REP 68 protein expressed by a host cell that expresses wild-type REP 78 or REP 68 protein under regulatory control of an AAV REP 78 or REP 68 promoter.
Dependencies are inferred from the claim text.