Sciences and Mathematics, College of
Biology, Department of
BURS Faculty Advisor
Dr. Felicity Sterling
Hurler/MPS-1 is a rare genetic disorder and a type of lysosomal storage disorder impacting the body’s ability to break down mucopolysaccharides due to a lack of enzyme alpha-L-iduronidase. As a result, the complex sugars accumulate in the body's tissues causing damage to various organs and tissues. There is currently no cure for MPS-I, but various treatments are available to manage its symptoms and increase the quality of life. Treatments include enzyme replacement and bone marrow transplantation which provide the body with a way to manage the symptoms and added health issues of this disease. In recent studies, aldurazyme has been approved as the first ERT, developed using recombinant DNA technology, for the treatment of MPS-I. In this research, the study focused on inducing MPS-I lysosomal tubules with the GAG antagonist, surfen. The cells were compared to show a significant difference when comparing the diameter and distribution of lysosomal tubules using immunofluorescence microscopy and cell imaging. It was seen that the lysosomal tubules with induced small molecules were effective in clearing GAG storage in the lysosome membrane tubules. Altogether, this research will allow room for more studies to explore the possibility of finding additional ways to cure Hurler. Future research should look at the synergistic approach and explore its effect on adverse side effects of GAG antagonist drugs.
Lodhi, Tibyaan, "Testing Small Molecules to Induce GAG Storage" (2023). Belmont University Research Symposium (BURS). 318.