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.