PARENT SESSION
1:30 PM to 3:30 PM
Sunday, April 22, 2001
Poster Session 07 Gene Therapy
Room: Exhibition Center

(P07-90) A Novel Complete Adenovirus Based Approach for recombinant Adeno-associated Virus Production.

Zhang, Xiuwu¹, Li, Chuan-Yuan¹, ¹

ABSTRACT-
Recombinant adeno-associated virus (rAAV) vectors have distinct advantages as a vehicle for therapeutic gene delivery. For example, rAAV can transduce heterologous genes efficiently into both dividing and non-dividing cells; it can infect multiple cell types such as the central nervous system muscle, lung, gut, liver, and eye; it causes minimal inflammatory responses and can mediate gene expression for over 1 year. There is also evidence indicating the initial signs of clinical success for rAAV-mediated hemophilia treatment. However, current methods for rAAV production are still too costly for clinical applications. In this study, we report a novel approach for rAAV production that is exclusively based on the adenovirus vector, without the need for plasmid transfections and special packaging cell lines. All components required for rAAV production, including the rep and cap genes, and the therapeutic gene(s), are delivered to the widely used 293 packaging cell line by adenovirus vectors. High titer rAAV vectors (200-600 Infectious units/producer cell) were obtained by use of this approach. As adenovirus vectors can be made to high titers and they can infect cells in suspension efficiently, this approach should be amenable for scaled-up production of rAAV vectors.

KEYWORDS: Adeno-associated virus, adenovirus, gene therapy