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Adeno Associated Virus

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Adeno-Associated virus (AAV) is a non-pathogenic helper dependant parvovirus.  This is one of the most promising vehicles for gene delivery. Recombinant AAV vectors have predominantly episomal gene expression. AAV has long term expression in terminally differentiated cells. It can infect a wide range of cells, including non-dividing cells. The major disadvantage of the AAV vectors is the small genome, which limits the size of the transgene to about 4.5 Kb.

Due to the toxic nature of the Rep proteins, and some of the adenovirus proteins, a packaging cell line is not available, making viral preparation more laborious. The Gene Transfer Vector Core uses the Baculovirus System to produce AAV. This system was developed by Dr. Robert Kotin at the National Institute of Health. This system is described in the following publication: “Insect Cells as a Factory to Produce Adeno-Associated Virus Type 2 Vectors” Human Gene Therapy 13:1935-1943 (November 1, 2002).

The Gene Transfer Vector Core offers the following AAV serotypes, AAV2, AAV2/1, AAV2/2, AAV2/4, AAV2/5, AAV2/8 and AAV2/9.

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