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Gene Therapy

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

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  1. Gene Therapy Correcting defective genes

  2. 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

  3. 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

  4. 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

  5. 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

  6. The future • Gene therapy on sex cells of carriers • Gene therapy on fertilised egg cells © 2010 Paul Billiet ODWS

  7. 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

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