Gene therapy

• Gene therapy

  • insertion/alteration of a functional allele
  • treatment for genetic disorders
• Somatic cell gene therapy

  alteration of alleles in body cells - those that are mostly affected by the disorder
• Somatic cell gene therapy

  • not a final cure
  • continuous treatment overtime
  • if treatment is stopped, eventually treated cells will die
  • potential side effects from method of gene delivery
• Germ line cell gene therapy

  alteration of alleles in sex cells
• Germ line cell gene therapy

  • one treatment, a cure
  • all cells are treated and will be passed onto offspring
  • any side effects cannot be reversed and will be passed onto offspring
• Benefits

  • prolongs lives
  • better quality of life
  • decreases number of those who suffer
• Drawbacks

  • may be used outside of medical treatment
  • expensive - money and resources may be more useful elsewhere
• drawbacks

  • potential immune risk
  • cancer risk - if mistakes are made
  • inserted allele may become over expressed
  • may be difficult to insert alleles into specific body cells
• IF there are two recessive alleles

  a dominant allele should be added
• IF there is a dominant allele

  it can be ‘silenced’ by sticking extra DNA within the allele so it does not work