problems with gene theropy

problems with gene treatments

How gene therapy works for cystic fibrosis

  • cystic fibroses occurs in a person with a critical gene sequence missing
  • the specific gene is placed inside a specially modified virus
  • the virus carries the missing gene sequence into the lungs cell
  • hopefully the cell will start producing the correct CFTR proteins and so remove the symptoms of CF
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Problems - extracellular barrier

  • there are Barriers before the new DNA in the vectors can reach the epithelial cells of the lungs
  • in CF the airways are coated with a thick mucus which prevents the sprays from penetrating lungs - one way to help is to dilute the mucus making it thinner
  • others are looking at a new root for the vector throught the blood to the lungs
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Problems - intracellular barriers and keeping the

  • once the vector is inside the cell it is a challenge to get the DNA  in the right  place to be transcribed
  • even when the new gene is taken in and transcribed it isn't perminent about two weeks at best
  • to try expending this period scientists are looking at different vectors aswell as looking at ways to repair the faulty allele on the original DNA (stem cells might lead to a cure)
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