๐๐ก๐๐ญ ๐๐ญ๐ญ๐ซ๐๐๐ญ๐๐ ๐ฒ๐จ๐ฎ ๐ญ๐จ ๐ญ๐ก๐ ๐๐๐ข๐๐๐ซ๐ญ ๐๐๐ก๐จ๐ฅ๐๐ซ๐ฌ ๐ฉ๐ซ๐จ๐ ๐ซ๐๐ฆ?
Research has always been part of my life. Growing up as a professorโs kid who valued research, I always knew it was something that I wanted to pursue when I grew up and went to college. As the first semester of my freshman year was ending, I had been part of two different research labs and knew I wanted to continue to do it for the semesters to come. When Dr. Larsen sent the Seifert Scholars application to the lab group, I immediately read about the scholarship and knew it was something I would want to apply for. Family brought me into the research world, and the Seifert family is allowing me to continue throughout this summer.
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๐๐ก๐๐ญ ๐ข๐ฌ ๐ฒ๐จ๐ฎ๐ซ ๐ซ๐๐ฌ๐๐๐ซ๐๐ก?
GM1 gangliosidosis is a rare, but fatal, inherited neurodegenerative disease. A lack or dysfunction of producing the lysosomal enzyme, ฮฒ-galactosidase, is caused by a mutation in the GLB1 gene and leads to the accumulation of normally degraded substrates. These substrates become stored in the lysosomes of all cells, accumulate, swell in the cytosol, and then die. This is particularly impactful in the central nervous system, as neurons die and can not be replaced, causing patient mortality at 2-4 years of age. The effectiveness of current treatments is limited due to the blood-brain barrier, which prevents transport of ฮฒ- galactosidase into the brain, eliminating the ability to increase the amount of enzyme to break down the accumulated substrates in the brain. Due to this challenge, there is no known treatment that slows down the progression of the disease or treats the most aggressive symptoms.
Through our research, we are developing emerging therapeutic approaches to deliver ฮฒ-galactosidase into the brain and enable its neurologic functions. This summer I am working to characterize the full biodistribution of these polymersomes with a focus on identifying specific cell types that are targeted. Ideally a significant portion of the polymersomes will deliver ฮฒ-galactosidase into neurons. In addition, this summer I am injecting GM1 affected mice with polymersomes with and without ApoE and determining their precise cellular location. The brains are isolated and collected for flow cytometry for cell sorting to determine the percentage of cells present in different brain cells including neurons, endothelial cells, and glial cells. Findings will be supported with immunofluorescence stains of brains sections. I am also running x-gal analysis and enzyme assays to confirm the spatiotemporal distribution of ฮฒ-galactosidase in the brain. These findings will increase our confidence in our therapeutic approach of GM1 gangliosidosis.
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๐๐ก๐๐ญ ๐ก๐๐ฌ ๐๐๐๐ง ๐ญ๐ก๐ ๐ข๐ฆ๐ฉ๐๐๐ญ ๐จ๐ ๐ญ๐ก๐ ๐๐๐ข๐๐๐ซ๐ญ ๐๐๐ก๐จ๐ฅ๐๐ซ๐ฌ ๐จ๐ง ๐ฒ๐จ๐ฎ๐ซ ๐๐๐ซ๐๐๐ซ?
The Seifert Scholars program allows me to focus all my time and energy on research. During the school year, my efforts are mostly focused on my classes, and research unfortunately has less of my attention. However, through this program, research is my focus this summer and the project that I am working on is directly related to the career path I want to take in the pharmaceutical industry.
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๐๐ก๐๐ญ ๐ข๐ฌ ๐ญ๐ก๐ ๐ง๐๐ฑ๐ญ ๐ฌ๐ญ๐๐ฉ ๐จ๐ ๐ฒ๐จ๐ฎ๐ซ ๐๐๐ซ๐๐๐ซ ๐๐จ๐ฅ๐ฅ๐จ๐ฐ๐ข๐ง๐ ๐ ๐ซ๐๐๐ฎ๐๐ญ๐ข๐จ๐ง?
While I am about to start my sophomore year at Clemson, I am still trying to decide
between staying in school for grad-school or going straight into the pharmaceutical industry. This summer has been helpful through the amount of research that I have been able to do to allow me to make my decision about my future career.