The major focus of my research group is to develop efficient approaches for gene delivery to the central nervous system (CNS), using recombinant adeno-associated virus vectors, to treat the neurological disease of Mucopolysaccharidosis (MPS) IIIB in patients. We also interested in mechanisms of post-transcriptional and post-translational modification of α-N-acetylglucosaminidase (the enzyme missing in MPS IIIB patients), which are critical for the function of the enzyme and the effectiveness of gene therapy. Another aspect of our research is understanding how the lysosomal storage of heparan sulfate, which is the primary consequence of the enzyme deficiency, activates different aspects of innate and adaptive immunity, and how the CNS immune response, especially the CNS T-cell autoimmunity, contributes to neuropathology and MPS IIIB disease progression. We are also implementing immune intervention strategies for therapeutic development for lysosomal storage diseases.