alevelssss
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Describe how a harmless virus, genetically engineered to contain a CFTR gene, can be used to insert the gene into a cystic fibrosis sufferer.

virus is inhaled / sprayed into the lungs;
gets into cells, inserting the healthy gene;
Thats the answer but how the hell would i know that little old me was just thinking it is inserted into plasmid to form recombinant dna?I know viruses act as vector so when faced with another question like this do i say the exact same thing???
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Jpw1097
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(Original post by alevelssss)
Describe how a harmless virus, genetically engineered to contain a CFTR gene, can be used to insert the gene into a cystic fibrosis sufferer.

virus is inhaled / sprayed into the lungs;
gets into cells, inserting the healthy gene;
Thats the answer but how the hell would i know that little old me was just thinking it is inserted into plasmid to form recombinant dna?I know viruses act as vector so when faced with another question like this do i say the exact same thing???
This is the classic example of gene therapy. I'm not really sure what you're asking. People with cystic fibrosis have two mutant alleles for the CFTR gene (since it's an autosomal recessive condition). Therefore, by inserting the faulty alleles with a normal CFTR gene, this will produce normal CFTR in the cell. The CFTR gene is inserted into a virus and can be administered via a spray. The virus is then taken up by the cells, the virus uncoats and the CFTR gene is released into the cytoplasm. The CFTR gene is then transcribed and translated to form the CFTR protein channe; which is then inserted into the membrane. This allows chloride ions to move into the secretions which draws water into the secretions by osmosis, making the secretions less thick and sticky. This only restores the respiratory function though, pancreatic secretions will still be thick and sticky. Also, the respiratory epithelium has a high turnover rate, cells are constantly being replaced and therefore cells containing the normal CFTR gene would also be replaced and therefore this would have to be administered regularly.
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alevelssss
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(Original post by Jpw1097)
This is the classic example of gene therapy. I'm not really sure what you're asking. People with cystic fibrosis have two mutant alleles for the CFTR gene (since it's an autosomal recessive condition). Therefore, by inserting the faulty alleles with a normal CFTR gene, this will produce normal CFTR in the cell. The CFTR gene is inserted into a virus and can be administered via a spray. The virus is then taken up by the cells, the virus uncoats and the CFTR gene is released into the cytoplasm. The CFTR gene is then transcribed and translated to form the CFTR protein channe; which is then inserted into the membrane. This allows chloride ions to move into the secretions which draws water into the secretions by osmosis, making the secretions less thick and sticky. This only restores the respiratory function though, pancreatic secretions will still be thick and sticky. Also, the respiratory epithelium has a high turnover rate, cells are constantly being replaced and therefore cells containing the normal CFTR gene would also be replaced and therefore this would have to be administered regularly.
okay that makes so much sense thank you for the explanation. However, I'm not sure that we have to know all of that for the aqa a2 spec, I'm pretty sure I've never even seen cystic fibrosis mentioned in our text book??
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Jpw1097
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(Original post by alevelssss)
okay that makes so much sense thank you for the explanation. However, I'm not sure that we have to know all of that for the aqa a2 spec, I'm pretty sure I've never even seen cystic fibrosis mentioned in our text book??
I went into probably more detail than you need. That does surprise me though, cystic fibrosis is the classic autosomal recessive condition.
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macpatgh-Sheldon
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How much you need to know depends on the examining board - a few years ago, the Nuffield-Salters A level biology syllabus required even more detail on cystic fibrosis than very aptly provided by JPW above.
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alevelssss
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Yeah I do Aqa so I don’t think it’s required but it can probably come up as a application question so getting a bit of context was still very useful
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