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Could CRISPR be used to fix this?


This kind of point mutation is exactly the kind of issue that CRISPR is capable of fixing. This can be done today. The issues are specificity and delivery of the fix. Delivery is hard to do non-invasively without side effects, and the 'fix' must not only correct only the error, but also not 'correct' a non-error elsewhere. Getting the 'patch' to the right place, and making sure the patch only affects exactly this sequence is now the hard part - actually creating the patch is doable today.


Exactly what I was thinking... but when will you (and society) be comfortable with a therapy that goes into your brain cells and edits their DNA?


>but when will you (and society) be comfortable with a therapy that goes into your brain cells and edits their DNA?

Seconds after some early adopters have shown that it works and has no side-effects?


No on will disagree with that answer.

Lots of people will argue vehemently about what it takes to be certain that there are no side effects.


>No on will disagree with that answer.

I disagree; there'll probably be a bunch of people who will vehemently disagree with "artificially" altering humans like this, even just to correct a genetic defect. They'll say it's "unnatural", or that it "isn't God's will", or somesuch.

I don't know that they'll be a big portion of the population, but they'll be out there.


Note that I didn't say: "Seconds after some early adopters tried it", but "Seconds after some early adopters have shown that it works and has no side-effects".

Which could be a few decades after some early adopters tried it -- e.g. until they died in their 80s or 90s with perfect brain function...

So, seconds after that...


Right, that was my understanding.

I suppose my point was that kornork seemed to me to be talking about safety and in that context, saying you'd do it after it is shown to be safe isn't saying a lot.


Aha, I took the parent's comments to be about the ethical and/or existential questions.

After all we do various kinds of brain altering operations and take brain altering drugs. DNA is not our personality, just how our body is built.


:) I'll bite. "Seconds" is not nearly long enough for me to be confident there are no adverse effects. Deleterious effects from monkeying with genes can take years or decades to show up (via increased risk to certain cancers, etc.)


When the alternative is your mind falling apart, I'd expect quite a few people will risk the side effects. I certainly would.


I'm ready


Not an expert on the technique, but if it's possible to "edit" a gene, how would the altered gene be propagated to billions of neurons (assuming that's where the gene has effect)? The idea of modifying the gene in situ is a technical issue that would seem awfully hard to resolve.

Of course the whole idea is predicated on having sufficient knowledge of the gene's effect. The reported finding awaits replication, and quite likely further refinement before practical applications are feasible.


I would assume editing In Situ would require a retrovirus or something similar. Seems incredibly risk, but then again, so was open-heart surgery until we figured out all the things that will go wrong.


There are more cells in a body than stars in the universe; I'm under the impression that the first CRISPR therapies will be on early-stage embryos where the number of cells is still manageable and we can feasibly edit all of them. Would be happy to hear more from someone who knows more about the latest in gene therapy though, my training here is getting a little dated.


1 x 10^24 stars vs 3.2 x 10^13 cells.

Estimates from a quick google.


Darn, internet info foiled by more internet info...




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