Gene therapy: would this be possible?

[GameChanger]

Hey there fellow Escapists, seeing as you guys are pretty intelligent, I figured you might want to help me out here:

I was studying for biology, (which is an actual separate subject in The Netherlands, not included in 'science') and one of the subjects was gene therapy. An example brought forward was the genetic malfunction called Cystic Fibrosis.

For those who don't know: cystic fibrosis is caused by a defection in the DNA which codes for a protein in the cellular membrane so that chloride-ions and sodium-ions can freely enter and leave the cells through active transport. The malfunction deforms this protein so that no longer happens. This causes secretions such as the fluids in the lungs to be very viscous and makes it harder to breathe, among other things. It affects vital processes, and patients with the disorder don't live very long.


A treatment could be gene therapy. Injecting the cells with a 'healthy' gene by means of viruses or liposomes. (artificially created particles consisting of membranes containing DNA)

The problem, of course, is that every cell in the body contains the defect gene. The treatment is not yet sufficient enough to literally change every cell in the human body.

But I've been thinking. Patients with cystic fibrosis can still have children. Would it be possible to cure the later generations of a patient by injecting their cells with the healthy gene in a very, very early stage in vitro? Like, stemcells, that early.

It would be great if someone with more knowledge could explain to me if this would work.

Feel free to correct any mistakes I've made.

PS. Please refrain from bringing politics and religion, I deliberately did not place this thread in that subforum, because this is a purely scientific question. Everyone who brings forward that "religion is stupid because they don't allow for stemcell research" will be mauled by a shoggoth, I mean it.

 

[Calibanbutcher]

Nope. Not possible.

You would have to change the DNA itself in the very first cells. After that it would porbably be too late.
Treat the sperm / oozyte and you might have a shot, though.

 

[GameChanger]

Nope. Not possible.
No matter.
You would have to change the DNA itself in the very first cells. After that it would porbably be too late.
Treat the sperm / oozyte and you might have a shot, though.

Yeah that's what I meant, inject the DNA in the very first stages, the fertilized egg in layman's terms.

 

[Calibanbutcher]

Nope. Not possible.
No matter.
You would have to change the DNA itself in the very first cells. After that it would porbably be too late.
Treat the sperm / oozyte and you might have a shot, though.

Yeah that's what I meant, inject the DNA in the very first stages, the fertilized egg in layman's terms.

Then I misunderstood what you meant.
That approach might be viable, though I believe only possible via in vitro fertilization..

 

[Esotera]

Seeing as the actual cause behind Cystic Fibrosis isn't really understood that well, the disease isn't a very good candidate for something as serious as gene therapy. There's also the issue that the CFTR gene has about 700 different variations that are implicated in cystic fibrosis, some of those variations might be completely different from each other which would make replacing the gene even more technically complex.

Anyway, what you're referring to is designer babies, or as Iain M Banks phrases it, genofixing. Genetically modifying a fertilised human egg cell wouldn't in theory be too much different from genetically modifying a bacterium cell. If you're interested in the specifics of how it works I can go into more detail, but basically you create a healthy copy of the gene, get a vector to carry it to the target cell, where it then has a small chance of replacing the faulty gene.

You can do this in fully grown humans, but there's quite a high risk of cancer & you'll never get all the target cells to take up the healthy gene. Also unless we can find a way to improve cell uptake of DNA, it'd be highly unlikely that the cell would actually take the healthy gene. I've attempted to create GM bacteria as part of my university course, the method is to try implanting it into millions of cells & select the hundred or so individuals where it successfully implanted and culture them.

 

[Korolev]

While we don't have the technology yet, I don't see why this can't be possible in the future. There are a raft of practical concerns when it comes to manipulating genes at that early a stage - how do you account for gene methylation, can you be sure that the genes are inserted in the right area, will the inserted genes be properly turned on and so forth, but I think that we'll be able to do this in about 40 to 50 years.

It would be a great way for people to have children and not worry about genetic defects. It would render any argument in favor of traditional eugenics completely mute (although this sort of technology could be considered a form of eugenics - nice eugenics, maybe).