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Gene Therapy - PowerPoint PPT Presentation
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Gene Therapy. Correcting defective genes. Definition & history. Normal gene inserted into the genome to replace non-functional gene Trials began in 1990 Cystic fibrosis gene moderately successful. © 2010 Paul Billiet ODWS. Vectors. Viruses eg retro viruses, adenoviruses (commonly used)
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Gene Therapy
Correcting defective genes
Definition & history
- Normal gene inserted into the genome to replace non-functional gene
- Trials began in 1990
- Cystic fibrosis gene moderately successful
© 2010 Paul Billiet ODWS
Vectors
- Viruses eg retro viruses, adenoviruses (commonly used)
- Direct introduction (“golden bullets”)
- Liposomes
- Endocytosis of DNA bound to cell surface receptors (low efficiency)
- Artificial chromosome (under development))
© 2010 Paul Billiet ODWS
Problems
- Acute immune response to viral vectors
- Repeated treatment neededGenes “lost” when the cell goes through mitosis
- Viral vectors could become pathogenic
- Genes spliced at random into the genome could upset other genes
- Multigene disorders too complex to treat
© 2010 Paul Billiet ODWS
Applications
- Curing genetic diseases
- Correcting cancer genes
- Inducing cancerous cells to make toxins so they kill themselves
- Blocking viral genes (e.g. HIV)
- Creating stem cells from somatic cells
© 2010 Paul Billiet ODWS
The future
- Gene therapy on sex cells of carriers
- Gene therapy on fertilised egg cells
© 2010 Paul Billiet ODWS
Ethical problems
- Gene therapy for serious genetic diseases OK but for other health problems?
- Somatic cell treatment stays with the individual, germ cell treatment passes down the germ line (becomes immortal)
- Very costly. Who pays? Who is eligible?
© 2010 Paul Billiet ODWS
