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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

Gene Therapy

Correcting defective genes

definition history
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
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
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
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
The future
  • Gene therapy on sex cells of carriers
  • Gene therapy on fertilised egg cells

© 2010 Paul Billiet ODWS

ethical problems
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