Gene Therapy

The research projects in Song’s group include: 1) improvement of safety and efficiency of rAAV vector by understanding molecular mechanisms of persistence and integration of the AAV genome in mammalian cells, and the immune responses to the transgene products; 2) the use of recombinant adeno-associated virus (rAAV) vectors mediated gene transfer to develop gene therapy approaches for common diseases such as alpha 1 antitrypsin (AAT) deficiency, diabetes and arthritis; 3) the development of adult stem cell based regenerative medicine; 4) functional studies for alpha 1 antitrypsin (AAT) and its therapeutic applications.