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Asked by andersone to Anne, Carolyn, Joe, Mariana, Nick on 18 Mar 2010 in Categories: General.
Question: What is your opinion on gene therapy for treating Cystic Fibrosis? To what extent do you think its affective?
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Nick Bradshaw answered on 18 Mar 2010:
Personnally I think it is a very good idea – but one which must be carefully tested to ensure that it is safe. Fortunatly a lot of work has already and is continuing to be done on that and it is looking good so far, with (as far as I am aware) no probelms so far. While I don’t like the idea of genetically engineering babies (not that I think it will happend any time soon) or anything like that, geneticly “tweaking” a few cells in a patient is to me a completly different matter. I gather quite a lot of CF patients seem keen on it.
As to whether it is efective – the theory appears good, and on paper it should work. Again, clinical trials are just beginning now, so hopefully we will have an answer in the next couple of years.
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Joseph Devlin answered on 18 Mar 2010:
Fraid I know nothing about this — couldn’t even guess. Sorry!
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Mariana Vargas answered on 18 Mar 2010:
Hello there, according to the Cystic Fibrosis trust website, they believe that gene therapy is the most likely cure for cystic fibrosis (http://www.cftrust.org.uk), and in fact this makes sense because the cause of the disease is a faulty gene which cannot produce the proper molecule for salt transport in cells, so replacing it with a healthy gene makes a lot of sense. Current research is being focused at delivering the healthy gene to the lungs, but initial trials need to establish the safety of this treatment first, and to find an appropriate way of delivering the gene to the patient’s cells.
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Carolyn McGettigan answered on 19 Mar 2010:
I think gene therapy for cystic fibrosis is currently pretty limited in effectiveness. From my reading, it seems that, while lab tests have shown great promise in importing corrective DNA strands into cells (via plasmids or viruses to carry the ‘correct’ sequence), the human tests have not gone less well and there have been problems in terms of how well the DNA gets transcribed and translated into protein. And it’s the proteins that are key to improving the airways in CF. I guess it’s just a matter of seeing how the methods develop.
Comments
Moderator - Sian commented on :
there is quite a controversial opinion that concludes gene therapy shouldn’t be carried out as defects are nature’s way of ensuring the survival of the fittest – how do you feel about that?
Joe commented on :
Ooh, going controversial on us – I like it. That make a strange argument because if you follow that logic, then medicine of any sort of pointless because you are twarting nature’s selection process. Certainly you wouldn’t immunize babies because if they don’t live through all the major diseases, then they aren’t strong enough.
Personally I think that’s crazy. We have a brain which allows us to not only survive in difficult circumstances but also to thrive. So we might as well use it to both improve our environment, and where necessary ourselves as well. Fixing parts of the body which are broken seems like a good use of our mental faculties to me.
Carolyn commented on :
I totally agree with Joe and Nick here. Also, carriers of one copy of the sickle cell anaemia mutation are resistant to malaria… Survival of the fittest isn’t always straightforward!
Nick commented on :
If someone did believe that genetic defects should be allowed to exist to ensure “survival of the fittest”, then it seems to be that they would have to also reject most other conventional medicines, after all you could argue that viruses and bacteria are also are more likely to kill people with “weaker genes”.
Fortunaly, as a society we believe in helping everyone, and not only the people who have the best genes.
I agree with Joe, evolution has given us a higher complex problem solving brain, it is perfectly right that we use it to improve our own health and ensure we survive.