Science University Research Symposium (SURS)

Utilizing Acarbose as a Model for Pompe Disease

Publication Date

Fall 11-24-2025

College

College of Sciences & Mathematics

Department

Biology, Department of

SURS Faculty Advisor

Felicity Sterling

Presentation Type

Poster Presentation

Abstract

Pompe Disease is a rare autosomal recessive disorder caused by mutations in the alpha-glucosidase (GAA) gene. These mutations leads to a pathological accumulation of glycogen in the lysosome, thereby impairing lysosomal function. At present, most studies involve patient cell lines and genetic manipulation. Both these methods are time consuming and low throughput. Therefore, we sought to identify another method to model Pompe Disease in cell culture. We identified acarbose, which is a competitive inihibitor of alpha-glucosidase, as a potential pharmacological model. To investigate its effectiveness as a model, we treated COS-7 and N27A cells with acarbose and observed changes to lysosomal morphology and function through immunofluorescent and live cell microscopy. Acarbose treatment mimicked what is seen in Pompe patient cell lines, thereby supporting our hypothesis that acarbose can be used for future investigations of Pompe disease.

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