Question: Technically, would germ-line therapy be an effective method to treat patients suffering from cystic fibrosis, and would this be a reliable method to ensure they would be cured in the long run?
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As cystic fibrosis is a genetically inherited disease, I think that germ-line therapy would prevent people from developing the disease. But this is not my area so I can only guess.
However, this quickly becomes an ethical issue with regard to genetic manipulation in humans. There is a really good film called GATTACA which is set in a futuristic world where genetic manipulation is common place. I highly recommend watching it!
My understanding is that germ line therapy is targeted to the cells which will be involved in the generation of sperm and ovules. This means that, it would prevent someone with the defective gene responsible of the cystic fibrosis from passing on this gene to his/her sons or daughters. However, I don’t think this can be used to cure someone who has been born with the disease.
I don’t think it can be used to treat someone who currently has the disease, but could allow a person with CF to not pass the mutation on to their child. The way CF is inherited is autosomal recessive, meaning that both parents have to contribute a CF mutation to the child, which they can do even if they don’t have CF themselves.
MaximDis – you ask interesting questions, always make have to look and read around for answers 😉
I don’t think this would cure the disease per say, but it would prevent the next generation from having it (given cystic fibrosis is an hereditary disease).
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