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Abstract #1150

Gene Transfer of Arginine Kinase to Skeletal Muscle using Adeno-Associated Virus

Sean C. Forbes1, Larry T. Bish2, Elizabeth R. Barton3, Fan Ye1, Celine Baligand4, H. L. Sweeney2, Glenn A. Walter4

1Department of Physical Therapy, University of Florida, Gainesville, FL, United States; 2Department of Physiology, University of Pennsylvania, Philadelphia, PA; 3Department of Anatomy & Cell Biology, University of Pennsylvania, Philadelphia, PA; 4Department of Physiology & Functional Genomics, University of Florida, Gainesville, FL

In this study we tested the feasibility of using 31P-MRS to monitor gene therapy by a commonly used nonpathogenic adeno-associated virus (AAV) delivery system. Muscle specific expression of the marker gene, arginine kinase (AK), was achieved using an AAV type 2/8 virus and the gene product (phosphoarginine) was monitored using 31P-MRS. The results indicate that AK was expressed within 8 weeks of delivery in muscle regions localized to the injection site. Therefore, delivery of AK gene via AAV may be effective as a reporter gene to noninvasively monitor the regional and global transfer of genes for therapeutic interventions.