As a postdoctoral trainee at Cincinnati Children’s Hospital Medical Center, I became inspired by my mentor and by work on patient-related projects. These experiences led me to my current research interest in the molecular pathogenesis of and treatments for lysosomal storage diseases.
In our lab, we are working to understand the pathogenesis of Gaucher disease and other lysosomal storage diseases so that we can develop specific therapies. My research interests include glycosphingolipids metabolism in neurodegenerative disease, specifically lipidomics and transcriptome to the relationship of Gaucher disease and Parkinson’s disease. We also study pharmaceutical small molecule therapy and brain organoid modeling of Gaucher disease. Our research is supported by the National Institutes of Health, foundations and industry-sponsored programs.
We investigate the therapeutic value of induced pluripotent stem cell-derived neural progenitor cells on Parkinson’s disease by increasing lysosomal acid β-glucosidase. We are also exploring the role of progranulin, a novel factor of acid β-glucosidase, as a potential therapeutic treatment of Gaucher disease.
Our recent published research work includes a 2019 study in Human Molecular Genetics outlining a noninvasive cell therapy for neurogenerative disease, and a 2020 study in EBioMedicine highlighting a new treatment strategy using a nanovesicles-based system for neuronopathic Gaucher disease.
The ultimate goal of my research is to translate discoveries into effective therapies for treating Gaucher disease and other neurodegenerative diseases.
Molecular pathogenesis and therapy of lysosomal storage diseases; glycosphingolipids metabolism; neurodegeneration.
Professor, UC Department of Pediatrics