Minimal Systems for Spatiotemporal Control of Gene Expression as Self-manufacturing Gene Therapeutics

The development of effective gene therapies is hindered by limitations in current delivery methods, such as the use of lipid nanoparticles (LNPs) and extracellular vesicles (EVs). We propose a novel approach to overcome these limitations by constructing a self-organizing minimal system capable of producing therapeutic mRNA. Our system leverages the power of lipid membrane compartments to control and regulate gene expression, mimicking the sophisticated spatiotemporal control found in living cells. Our research will focus on three main objectives: (1) investigating the activity of transcriptional and translational machinery in the proximity of lipid membranes, (2) designing and constructing a minimal system that exhibits spatiotemporal self-organization of gene expression, and (3) developing self-manufacturing platforms for the delivery and development of gene therapeutics. By combining expertise in chemistry, biology, physics, and engineering, we aim to create a highly reproducible and efficient system for producing therapeutic mRNA. This will pave the way for the development of safer, more targeted, and more effective gene therapies, ultimately revolutionizing the treatment of various diseases.