Gene Therapy Project: DNA Road Trip Tiffany Barbour, Mentor: Dr. Shelly Sakiyama-Elbert, Department of Biomedical Engineering, Washington University Gene therapy has the potential to treat genetic disorders, such as cystic fibrosis, and has applications for tissue engineering. The main objective of gene delivery is to deliver DNA to the nucleus of the cell. There are two types of gene delivery vehicles: viral and non-viral or synthetic. Viral vehicles are extremely effective in guiding their genetic materials to the nucleus; however, they pose safety risks and limitations. Synthetic vehicles include positively charged polymers such as Poly-L-Lysine (PLL) and Polyethyleminine (PEI), and liposomes. These synthetic vehicles, while safer, have significantly lower DNA delivery efficiency than the viral vehicles and are often toxic to cells due to their positive charge. Polyethylene glycol (PEG), a neutrally charged and biocompatible polymer, was used instead of PEI and PLL to create a less toxic vehicle; however, by itself it cannot bind DNA. By modifying the PEG to contain two acrylate groups, we were able to attach CBZIP, a DNA binding peptide. This CBZIP-PEG vehicle was able to deliver DNA to the nucleus with the same efficiency as PLL. Future work will improve this efficiency by increasing the number of acrylate groups on the PEG molecule and testing alternative DNA-binding peptides. Scholar Index Next   Natural Sciences Learning Center Washington University - Biology All contents copyright © 2004