RESEARCH INTERESTS

Improvement of safety and efficiency of recombinant Adeno-Associated Virus (rAAV) vector by understanding molecular mechanisms of persistence and integration of the AAV genome in mammalian cells. Current research focuses on the effect of DNA dependent protein kinase (DNA-PK) on rAAV genome persistence and integration.

The use of rAAV vectors mediated gene transfer to develop gene therapy approaches for common diseases such as diabetes. Current research projects are anti-inflammatory serpin gene transfer for enhancing islet transplantation and for preventing type 1 diabetes in NOD mouse model, and gene therapy for alpha 1 antitrypsin gene deficiency.

• AAV biology and vector construction
• Gene Delivery
• Molecular and cellular biology
• Gene expression and regulation

• rAAV mediated gene transfer

1. Song S., Scott-Jorgensen M., Wang J., Poirier A., Crawford J., Camphell-Thompson M., Flotte T.R.  “Recombinant Adeno-associated Virus 2-alpha 1-antitrypsin (rAAV-AAT) Vectors in a Non-human Primate Model: Safety and Immunologic Aspects.“  Molecular Therapy, 6(3):329-335 (2002).
2. Mah C., Fraites T.J., Zolotukhin I., Song S., Flotte T.R., Batich C., Byrne B.J.  “Improved Method of Recombinant AAV2 Delivery for Systemic Targeted Gene Therapy.”  Molecular Therapy, 6(1):106-112 (2002).
3. Goudy K., Song S., Wasserfall C., Zhang Y.C., Kapturczak M., Muir A., Scott-Jorgensen M., Campbell-Thompson M., Crawford J.M., Ellis T.M., Flotte T.R., Atkinson M.A. “Adeno-associated Virus Vector-mediated IL-10 Gene Delivery Prevents Type 1 Diabetes in NOD Mice.”  Proc. Natl. Acad. Sci. USA 98(24):13913-13918 (2001).
4. Song S., Laipis P.J., Berns K.I., Flotte T.R.  “Effect of DNA-PKcs on the Molecular Fate of  the rAAV2 Genome in Skeletal Muscle.”  Proc. Natl. Acad. Sci. USA, 98(7):4084-4088 (2001).
5. Song S., Embury J., Laipis P.J., Berns K.I., Crawford J.M., Flotte T.R.  “Stable Therapeutic Serum Levels of Human Alpha-1 Antitrypsin (AAT) After Portal Vein Injection of Recombinant Adeno-associated Virus (rAAV) Vectors.”  Gene Therapy, 8:1299-1306 (2001).