October 17, 2014
Conducting Scientific Research as a First-Year Student: Analysis of Small Molecules as Pharmacological Chaperones of NEU1
As a first-year biochemistry student, I applied to research opportunities across the country and received an acceptance from the Institute of Cellular Engineering Research Experience for Undergraduates (ICE REU) program at UMass, Amherst. For nine weeks, I collaborated with a postdoc in Professor Scott Garman’s lab, delving into various aspects of research, such as designing experiments and reading scientific papers. We quantified the effects of two small molecules, NANA and DANA, acting as pharmacological chaperones of NEU1, which is the deficient enzyme in the lysosomal storage disease, sialidosis. NANA and DANA help stabilize the mutated protein so it can be trafficked to the lysosome and prevent disease symptoms. In order to do this, I had to learn protocols to purify wild-type protein, test the stabilizing effect of these small molecules, and show their effectiveness in a cellular system. This school year, I am picking up where I left off over the summer and am working on designing new experiments to bring this phase of the project to a conclusion. Through this experience, I discovered that research is something I really enjoy and I am excited to explore all the possibilities within this field.
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