Transcript non-viral gene therapy - POKEWEED-ANTIVIRAL

NON-VIRAL GENE
THERAPY
Amanda Aryee
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.
History
Knowledge in Gene Therapy
10
9
8
Arbitrary Units
7
6
5
4
3
2
1
0
Time (years)
• 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.
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)
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.
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.)
SUMMARY AND CONCLUSION
Advantages of non- viral gene therapy include:
• Biocompatibility (Low toxity and nonimmunogenicity)
• 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.