Dwight Koeberl, MD, PhD of Duke University School of Medicine explains the difference between enzyme replacement therapy (ERT) and gene therapy, as it relates to lysosomal storage disorders like Pompe disease.
Dr. Koebel is currently conducting a gene therapy clinical trial for Pompe disease. To see his overview of that study, click here.
Pompe disease is a lysosomal storage disorder in which mutations in the GAA gene leads reduced levels of the GAA enzyme. The net result is a buildup of glycogen in the body’s cells that can cause to numerous organs, most notably muscles.