An Investigation Into Factors Affecting Ultrasound and Microbubble Mediated Gene Transfection

摘要: Gene therapy is a promising tool in the treatment of genetic disorders. Viral vectors have a natural ability to genetically alter cells, however, concern over their immunogenic and mutagenic effects has led to the development of safer non-viral alternatives, such as ultrasound and microbubble mediated gene transfection (UMGT). UMGT causes cellular bioeffects leading to uptake of genetic material; however transfection efficiencies in vivo are poor. It was the aim of this project to investigate key factors that affect UMGT in vivo. A Model Protocol was created with techniques capable of characterising the acoustic response and stability of microbubbles, as well as their population statistics and ability to transfect the heart of six to eight week old, CD1, female mice with a luciferase plasmid. This was used to investigate the effect of needle gauge and microbubble formulation on transfection.