Overview
My overall research interests are finding effective treatment for human glycogen storage diseases (GSDs) and other inherited metabolic disorders. My current research focuses on identification of novel therapeutic targets and development of effective therapies for GSD II (Pompe disease), GSD III (Cori disease), and GSD IV (Andersen disease) using cellular and 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
Gene therapy for glycogen storage diseases.
Journal Article J Inherit Metab Dis · January 2024 Glycogen storage disorders (GSDs) are inherited disorders of metabolism resulting from the deficiency of individual enzymes involved in the synthesis, transport, and degradation of glycogen. This literature review summarizes the development of gene therapy ... Full text Link to item CiteSuccessful AAV8 readministration: Suppression of capsid-specific neutralizing antibodies by a combination treatment of bortezomib and CD20 mAb in a mouse model of Pompe disease.
Journal Article J Gene Med · August 2023 BACKGROUND: A major challenge to adeno-associated virus (AAV)-mediated gene therapy is the presence of anti-AAV capsid neutralizing antibodies (NAbs), which can block viral vector transduction even at very low titers. In the present study, we examined the ... Full text Open Access Link to item CiteSuppression of pullulanase-induced cytotoxic T cell response with a dual promoter in GSD IIIa mice.
Journal Article JCI Insight · December 8, 2022 Glycogen debranching enzyme deficiency in glycogen storage disease type III (GSD III) results in excessive glycogen accumulation in multiple tissues, primarily the liver, heart, and skeletal muscle. We recently reported that an adeno-associated virus vecto ... Full text Open Access Link to item CiteRecent Grants
Gene 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 - 2026CHARACTERIZATION OF GSD IX ¿2 and GSD VI MOUSE MODELS
ResearchInvestigator · Awarded by Kriya Therapeutics · 2022 - 2024View All Grants
Education, Training & Certifications
China Medical University (China) ·
1995
Ph.D.