Using E. coli to develop alternative treatment for NKH.
Our project aims to develop an alternative treatment for Nonketotic Hyperglycinemia (NKH) patients that are more accurate. We hope that with our product, patients can have fewer side effects and an easier way to get medicated and thus achieve a more convenient life. Thus, people who are affected by NKH are our proposed ultimate end users. However, the immediate end users would be medical research scientists who would need to test each intermediate for safety and efficacy beginning in cell culture. The process leading to FDA approval is long and expensive, which is out of our ability in many ways. We hope that after all the testing and approval, our product can be produced in factories where both safety measures and production efficiency are obtained with the help from the model created by the William and Mary iGEM team.
Safety-wise, since we use E. coli as part of our production method, we need to consider the possibility of E. coli contamination of the end products. To solve such problems, we would design a multi-step production system, decontamination measures, and checkpoints. Bacteria are dangerous when alive, but the final product would be safe once the E. coli is killed, broken down, and filtered out during decontamination. Even more, ideally, the production would occur in a cell-free system using the enzymes produced by the modified E. coli and then purified. As the product is more mature and gets into factory production, the prices of the products would be lowered.