Fu lab is a translational biomedical research lab. The 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. Our research 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. Currently, we have expanded our research efforts to develop effective gene therapy products to treat broad lysosomal storage diseases (MPS I, MPS IIIC, MPS IIID, NPC1, Krabbe, ML) and other neurogenetic disorders, while making new generation AAV vectors with improved efficacy for MPS IIIA, IIIB and II.
Our research also targets the critical challenge in translating rAAV gene therapy to clinical application, the widespread pre-existing antibodies against AAV in humans. We have demonstrated that effective antibody depletion requires broad immunomodulation and are testing a novel transient antibody clearance approach for AAV gene delivery. Further, the blood-brain-barrier (BBB) has been a major obstacle in therapeutic development for neurological diseases. To address this challenge, we are working towards develop novel techniques facilitating/enhancing AAV gene delivery to the central nervous system (CNS) across the BBB.
Finally, lack of effective biomarkers for rare neurogenetic diseases poses challenges to the translation of gene therapy to clinic. Therefore, we have made efforts to identify biomarkers, especially neuro-specific biomarkers for neuropathic lysosomal storage diseases, using transcriptional profiling and metabolomics. The mission of Fu Laboratory is to develop and translate gene therapy products to treat neurogenetic diseases in humans and make effective gene therapy available to all patients in need.