8 years ago
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Gene therapy treatment for cystic fibrosis may be possible by 2020, scientists say
Although results of first trial were ‘modest and variable’, second bigger trial will combine gene therapy with other treatments for longer term benefits
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http://www.theguardian.com
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Why this is very cool.. Cystic fibrosis is caused by a faulty gene. So that basically leaves you with few options:
-either fix the gene - we have the technology but it's not safe enough to use in patients right now
-deliver a new, working copy of a gene - they would use viruses for that, which is not ideal
-or, what they have done here - deliver DNA circles with the gene without the need for viruses
I think that both using viruses or fixing genes would have better results, but we need more time to develop these therapies. So right now those scientists have come up with using DNA circles. And, according the results of the study, it's sort of working.. So they made DNA, gave them to patients and voila - gene therapy for cystic fibrosis is not fiction anymore. Give it another twenty years and we will be fixing the faulty genes instead :)
I've read through the original lancet article (well, skimmed through it to be honest) and it seems to be a small effect. Significant but on the lower end of the spectrum. Nevertheless that is some exciting development. I'm particularly impressed by the vector used. Plasmids encapsuled in liposomes is a pretty great idea. Especially since virus vectors do not work very well in the lungs due to very strong immunoactivity. I really hope that further trials can build on that and maybe even improve FEV (forced expiratory volume, a measurement of how much air you can exhale in 1s) instead of just stabilizing it.
Yeah, you're right. It's a good start though - I imagine they had to play it safe just to establish any side effects etc.