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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