AAV-2 is a single-stranded DNA parvovirus which has recently emerged as potentially useful tool for gene transfer, both ex vivo and in vivo. We had previously found that a recombinant AAV-2 vector which expresses the beta-galactosidase gene was able to transduce greater than 90% of both primary and immortalized human fetal glial cells in vitro at an MOI of between 5-10. Furthermore, a rAAV was also able to efficiently transfer, integrate and stably express the c-DNA for the human CD4 molecule into the immortalized human astrocyte cell line SVG. This new cell line, SVG-CD4, was found to support a productive persistent infection of the lentivirus HIV-1, demonstrating that the AAV can transfer a functional transgene as well. Recent work has studied the potential use of rAAV vectors as vehicles for therapeutic gene transfer in vivo, and expression following stereotaxic injection of the recombinant vector into the spinal cord of normal Sprague-Dawley rats. RAAV, which expresses the beta-galactosidase gene, successfully transduced a relatively large number of cells in the grey matter of the midthoracic spinal cord for up to 14 days postinjection.

Agency
National Institute of Health (NIH)
Institute
National Institute of Neurological Disorders and Stroke (NINDS)
Type
Intramural Research (Z01)
Project #
1Z01NS002915-03
Application #
6163103
Study Section
Special Emphasis Panel (LMMN)
Project Start
Project End
Budget Start
Budget End
Support Year
3
Fiscal Year
1997
Total Cost
Indirect Cost
City
State
Country
United States
Zip Code