Correcting defective genes

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Presentation transcript:

Correcting defective genes Gene Therapy Correcting defective genes © 2016 Paul Billiet ODWS

Definition & history Normal gene inserted into the genome to replace non-functional gene Trials began in 1990 Cystic fibrosis gene moderately successful © 2016 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)) © 2016 Paul Billiet ODWS

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 © 2016 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 © 2016 Paul Billiet ODWS

The future Gene therapy on sex cells of carriers Gene therapy on fertilised egg cells © 2016 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? © 2016 Paul Billiet ODWS