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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.

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Presentation on theme: "Gene Therapy Correcting defective genes. Definition & history  Normal gene inserted into the genome to replace non-functional gene  Trials began in."— Presentation transcript:

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 ODWSODWS

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 ODWSODWS

4 Problems  Acute immune response to viral vectors  Repeated treatment needed Genes “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 ODWSODWS

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 ODWSODWS

6 The future  Gene therapy on sex cells of carriers  Gene therapy on fertilised egg cells © 2010 Paul Billiet ODWSODWS

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 ODWSODWS

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