| 摘要 |
PROBLEM TO BE SOLVED: To provide 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, and to provide assessment of the screening of agents that affect viral infectiveness and/or replication.SOLUTION: At the onset of vector production, AAV producer cells 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 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 are substantially free of helper virus as well as helper viral proteins and cellular proteins and other contaminants. |
