Engineering Synthetic Insulator Elements for Gene Therapies

This project will create new biological parts for gene therapies. Specifically, we will utilise a synthetic biology approach (design-build-test-learn) to engineer novel constructs that permit long-term episomal expression in a wide range of human cell-types in vivo. Context-specific design criteria (e.g. minimal/maximal immunogenicity, minimised burden on cellular chassis machinery, etc.) will be built into these parts to create constructs that are fully optimised for this high-value application. Parts exhibiting these functionalities also have significant potential in other biotechnology fields, particularly transient production of recombinant protein products. Accordingly, a library of parts will also be tailored specifically for use in these biopharmaceutical manufacturing systems. Engineered parts will be 'reduced to practice' by exemplifying their utility in bioindustrial systems.