1. NON-VIRAL GENE THERAPY Amanda Aryee

2. Gene Therapy Gene therapy is the process of introducing foreign DNA into host cells. Gene therapy has the potential to bring about tremendous changes in treatment of various diseases and disorders. The therapy relies on gene replacement as the solution to treat the disorders.

3. History 1953 Watson and Crick discovered DNA model. The concepts of chromosomes, genes, etc. developed thereafter. 1990’ the first person was treated. White blood cells were extracted from the body.

4. Methods of Gene Therapy VIRAL GENE THERAPY Virus mediated gene delivery utilizes ability of a virus to inject its DNA inside a host cell. A gene that is intended for delivery is packaged into a viral particle. NON-VIRAL GENE THERAPY Micro-injection Gene gun Impalefection Hydrostatic pressure Electroporation Continuous infusion Sonication Chemical e.g. lipofection. It can also include the use of polymeric gene carriers (polyplexes)

5. ELECTROPORATION A significant increase in the electrical conductivity and permeability of the cell plasma membrane caused by an externally applied electrical field ( 300-400 mV for < 1 ms membrane span). It is used primarily to help get genes and drugs into cells. Acts on the cell membrane, leaving collagen fibres and other vascular tissue structures intact. Allows healthy cells to re-grow far more quickly.

6. ELECTROPORATION Schematic showing the theoretical arrangement of lipids in a hydrophobic pore (top) and a hydrophilic pore (bottom). An electrode used for irreversible electroporation, which could soon be used to treat tumors in humans. (Image courtesy of Oncobionic Inc.)

7. SUMMARY AND CONCLUSION Advantages of non- viral gene therapy include: Biocompatibility (Low toxity and non- immunogenicity) Efficient nucleic acid delivery and manufacture in commercially acceptable form. There have been significant improvements made over the past few years that need to be integrated into current systems to exploit their combined attributes.