https://orcid.org/0000-0002-2191-0025
Overview
My overall research interests are finding effective treatment for human inherited metabolic disorders, including glycogen storage diseases (GSDs) and lysosomal storage diseases (LSDs) . My current research focuses on identification of novel therapeutic targets and development of effective therapies for Pompe disease (GSD II) and Cori disease (GSD III) using murine and canine 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.
Current Appointments & Affiliations
Associate Professor in Pediatrics
·
2021 - Present
Pediatrics, Medical Genetics,
Pediatrics
Recent Publications
Novel promoters drive therapeutic transgene expression and evade transgene-specific immune responses in a mouse model of Pompe disease.
Journal Article Mol Genet Metab · March 2026 Systemically administered gene therapy is under development for the treatment of Pompe disease, an inherited lysosomal storage disorder caused by acid alpha-glucosidase (GAA) deficiency. We evaluated recombinant AAV9 vectors expressing GAA driven by the mi ... Full text Link to item CiteHigh-potency MyoAAV capsids enhanced skeletal muscle correction in a mouse model of GSD IIIa.
Journal Article Mol Ther Methods Clin Dev · September 11, 2025 Glycogen storage disease type IIIa (GSD IIIa) affects multiple tissues, including liver, heart, and skeletal muscles. We recently reported that an adeno-associated virus serotype 9 vector expressing pullulanase, a bacterial glycogen debranching enzyme, dri ... Full text Open Access Link to item CiteAAV Gene Therapy with a Bacterial Glycogen Debranching Enzyme in a Dog Model of GSD IIIa
Conference MOLECULAR THERAPY · April 28, 2025 Link to item CiteRecent Grants
Skeletal muscle-targeted gene therapy for Pompe disease
ResearchPrincipal Investigator · Awarded by National Institutes of Health · 2025 - 2030Gene therapy for glycogen storage disease type III
ResearchPrincipal Investigator · Awarded by National Institute of Arthritis and Musculoskeletal and Skin Diseases · 2022 - 2027Stable therapy in Pompe disease through genome editing
ResearchCo Investigator · Awarded by National Institute of Arthritis and Musculoskeletal and Skin Diseases · 2021 - 2026View All Grants
Education
China Medical University (China) ·
1995
Ph.D.