abstract |
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 beginning of vector production, the AAV producer cell contains one or more AAV packaging genes, an AAV vector containing a heterologous (ie, non-AAV) transgene of interest, and a helper virus such as an adenovirus. Including. The produced AAV vector preparation can mediate delivery of the transgene of interest (eg, a therapeutic gene) to any wide range of tissues and cells. The AAV vector preparation is also substantially free of helper virus, and helper virus proteins and cellular proteins and other contaminants. Quantitative, high-throughput assays that are useful in assessing screening for agents that affect viral infectivity and / or replication. [Selection figure] None |