Research Interests
My major research focus has been to develop effective gene delivery approaches to target the entire nervous system, using adeno-associated virus (AAV) vectors, for treating neuropathic lysosomal storage diseases in humans. These efforts have led to three IND approvals of Phase 1/2 gene therapy clinical trials in patients with mucopolysaccharidosis (MPS) IIIA, MPS IIIB and MPS II using trans-BBB-neurotropic AAV9 vectors via systemic delivery. My team expand our efforts towards 1) developing effective gene therapy targeting broad neurogenetic diseases, 2) developing novel approaches to improve and enhance the CNS gene delivery across the BBB, 3) exploring novel agents and approaches for effective antibody depletion and 4) identifying effective biomarkers for neurological diseases. My goal is to translate effective gene therapy products to treating neurogenetic diseases in humans and improve the quality of life of patients and their families.
