Research Interests

AAV vectors have been successfully applied in clinical trials in patients with eye and liver diseases. Several challenges limit AAV application including the capsid specific CTL response, AAV neutralizing antibody presence in human population, late innate immune response after AAV transduction and low transduction efficiency of AAV vector in humans. In light of our most recent works on AAV vector immunology and AAV delivery, the core research theme is to explore novel strategies for development of AAV vectors with the ability to target human tissues and evade AAV immunity. My lab has mainly focused on following three major projects:

1) Study the immune response against AAV vectors, including a CTL response to the AAV capsid, AAV neutralizing antibodies, CTL response to transgenes, transgene inhibitors and innate immunity to AAV vectors.

2) Develop novel strategies to enhance AAV vector transduction. Several approaches has been explored including serum proteins and vector virion binding peptides.

3) Optimize AAV vectors for hemophilia gene therapy, especially for hemophilia with inhibitors.