A Novel Synthetic Biology Approach for MPRA-Optimized Protein Cloning to Stop the Progression of Tay-Sachs Disease

The objective of our project is to stop the progression of Tay-Sachs Disease through the extraction of cloned beta hexosaminidase-A from E.coli cells.
We intend on replacing the lac gene from the plasmid pUC19 with HEXA in order to overproduce the alpha subunit of β-HexA.
Additionally, we plan on determining protein production by using sodium dodecyl-sulfate polyacrylamide gel electrophoresis and a Western blot
Finally, we will use MPRA arrays to optimize the amount of protein produced and evaluate the presence of off-target effects to be able to mitigate them.
Some of our next steps include testing our solution in mammalian cells and evaluating various delivery techniques including a nasal spray and spinal injection.

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