Science University Research Symposium (SURS)
Publication Date
2024
College
Sciences and Mathematics, College of
Department
Biology, Department of
SURS Faculty Advisor
Felicity Sterling
Presentation Type
Poster Presentation
Abstract
Sanfilippo Syndrome (MPS III) is an autosomal recessive lysosomal storage disorder. This disease is prevalent in children at a young age. The gene that affects Sanfilippo is the HGSNAT gene which normally breaks down and recycles heparan sulfate, a large sugar molecule. Heparan sulfate accumulates and causes damage to the cells of the body, particularly in the brain and spinal cord by blocking normal lysosomal function. The buildup of heparan sulfate in brain and spinal cord cells leads to progressive neurological symptoms, including cognitive decline, movement disorders, and behavioral issues.
Previous work done in our lab showed that when we treat cells with PugNac we see an increased lysosome size and increase in mitochondrial fragments. The buildup of heparan sulfate enlarges the lysosome so it loses its function of clearing debris which will then kill the mitochondria. However this work has only been done in fibroblasts. We repeated the same immunofluorescent and live microscopy experiments in N27A cells to see if the same effect can be seen on neurons.
When the cells were dosed with PugNac, the lysosomes became much larger than usual, which suggests that their normal function is affected. For the mitochondria, there were many small fragments, and a larger number of mitochondria appeared dead rather than alive, indicating they were not functioning properly.
Recommended Citation
Salib, Mariam M. and Tadros, Emily A., "Effects of PugNac Treatment on Lysosomal and Mitochondrial Function in N27A Neurons" (2024). Science University Research Symposium (SURS). 192.
https://repository.belmont.edu/surs/192
