Date Wednesday, May 30, 2012
Time: 12 p.m. to 1 p.m.
Venue: Oborowsky Degner Seminar Hall
(1-040 LKS)
"Mechanistic studies on the uptake and intracellular trafficking of lipid-modified cationic polymers for gene delivery in primary cells"
Charlie Hsu
PhD candidate
Abstract:
Gene therapy is a promising approach to treat a wide range of acute, chronic and infectious diseases. The approach is based on the notion that genetic materials can be delivered to the target cells to correct physiological abnormalities at the genetic level. This process require an efficient "gene carrier" to facilitate targeted cellular delivery of the genetic cargo to defined intracellular compartments. Cationic polymers are among one of the most promising synthetic alternative to engineered viruses as delivery agent. But low precision and low efficacy currently hampers the translation of these non-viral gene carriers to clinical reality. In this thesis work, we examined several intracellular events that are critical to the eventual execution of the genetic instructions. Specifically, we looked at how the different shapes and sizes of DNA affects its uptake as well as how lipid modification alters the uptake pathways and intracellular routing of DNA/carrier complexes. This body of work should enhance our understanding of the intracellular fate of gene complexes and facilitate rational design of more effective gene carriers