Baodong Sun, PhD
My overall research interests are finding effective treatment for human glycogen storage diseases (GSDs) and other inherited metabolic disorders. My current research focuses on identification of novel therapeutic targets and development of effective therapies for GSD II (Pompe disease), GSD III (Cori disease), and GSD IV (Andersen disease) using cellular and animal disease models. The main therapeutic approaches we are using in our pre-clinical studies include protein/enzyme therapy, AAV-mediated gene therapy, and substrate reduction therapy with small molecule drugs.
Education and Training
- Ph.D., China Medical University (China), 1995
Selected Grants and Awards
- Gene therapy in GSD III dogs
- Immunosuppression to deplete anti-AAV antibodies
- Activity and Biodistribution of the AAV2/8-LSPhGAA in GAA-knockout mice
- Preclinical study of use of SVP-Rapamycin to induce immune tolerance to ERT in Pompe disease mice
- Evaluation of the use of Myozyme for treatment of glycogen storage disease type III and IV in murine disease models
- Liver-specific knockdown of M6PR to improve efficacy of ERT in muscle of Pompe disease mice
- In vivo efficacy studies of Fab-GAA treatment for GSDII, III, and IV in murine disease models
- To test the ability of SVP to prevent immune response in Pompe disease mouse model.