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Baby is healed with first personalized gene-editing treatment

nytimes.com

111–120 of 570 posts

Re: Baby is healed with first personalized gene-editing treatment

#111

Are the edited genes inherited, or the original ones? Does the previous question have an answer that depends on the babies sex? From an evolutionary perspective it‘s interesting how the further medicine gets, the more we inherit genes unfit for life without medical support.

couldn't be unless they reached reproductive cells.

Re: Baby is healed with first personalized gene-editing treatment

#112

> To accomplish that feat, the treatment is wrapped in fatty lipid molecules to protect it from degradation in the blood on its way to the liver, where the edit will be made. Inside the lipids are instructions that command the cells to produce an enzyme that edits the gene. They also carry a molecular GPS — CRISPR — which was altered to crawl along a person’s DNA until it finds the exact DNA letter that needs to be c…

> That is one of the most incredible things I have ever read.

This is even more great reading behind the above:

https://en.wikipedia.org/wiki/Jennifer_Doudna

Re: Baby is healed with first personalized gene-editing treatment

#113
post #83

> To accomplish that feat, the treatment is wrapped in fatty lipid molecules to protect it from degradation in the blood on its way to the liver, where the edit will be made. Inside the lipids are instructions that command the cells to produce an enzyme that edits the gene. They also carry a molecular GPS — CRISPR — which was altered to crawl along a person’s DNA until it finds the exact DNA letter that needs to be c…

One other fun part of gene editing in vivo is that we don't actually use GACU (T in DNA). It turns out that if you use Pseudouridine (Ψ) instead of uridine (U) then the body's immune system doesn't nearly alarm as much, as it doesn't really see that mRNA as quite so dangerous. But , the RNA -> Protein equipment will just make protiens it without any problems. Which, yeah, that's a miraculous discovery. And it was wel…

> [...] then the body's immune system doesn't nearly alarm as much, as it doesn't really see that mRNA as quite so dangerous

Please tell me there are measures to prevent this going into the wild. Please tell me this won't be used in large-scale industrial farming.

Re: Baby is healed with first personalized gene-editing treatment

#114
post #95

Are the edited genes inherited, or the original ones? Does the previous question have an answer that depends on the babies sex? From an evolutionary perspective it‘s interesting how the further medicine gets, the more we inherit genes unfit for life without medical support.

No, it's only in the liver, from what I can tell from the science, not the gametes. No, it would not depend on the sex of the baby, as the chromosomes that you're editing aren't X or Y. Evolutionarily, the inheritance of genes is a far slower process than the medical advancements we make, so what I think we're seeing here is a chasing down of the low probability events. In that, most of the evolutionary pressure is c…

If they could get complete delivery to the liver stem cells, then the change could be permanent, although this is making many simplifications.

Organs in your body usually keep some very old cells (formed in the embryo) around which act as parents for all the new cells in an organ. Any cell can only divide a limited number of times, so they typically maintain a "tree structure" where the old cells create children and grandchildren (etc) that then differentiate into the organ-specific cells that do the actual organ work.

If you modify only the differentiated cells, eventually they die, and are replaced by descendents of stem cells; if those stem cells didn't get modified, their descendents will not have the fix, and the treatment efficacy reduces over time.

Re: Baby is healed with first personalized gene-editing treatment

#118
post #115
post #87

[flagged]

Delete your comment. You're minimising the death of children. Yeah I'm sensitive about this. I like many other parents of children who have been lost to genetic disease have good reason to be.

We can pause and acknowledge that life (and what that means) will change, while also acknowledging that for many it will be the easiest decision in the world for it to change.

Re: Baby is healed with first personalized gene-editing treatment

#119
post #80

I think it was here a few years ago that I read a comment saying that sick children will be the Trojan horse for normalizing gene editing of humans, because who could say no to sick children, right? Well, guess it's here now, so how long utill the eugenics wars start?

it's unclear the outcome of this will be eugenics wars. Answering the real question- it's unlikely these techniques will see widespread "recreational" usage any time soon, as they come with a wide range of risks. Further, the scientific community has learned a lot from previous eugenics programs; anything that happens in the future will happen with both social and political regulation. It's ultimately hard to predict…

The problem won't be that there will be those who want to have babies with edited genomes, and those who oppose that.

It will be that people just don't have children at all.

Re: Baby is healed with first personalized gene-editing treatment

#120

Earlier quoted context omitted.

Yep, this effort is the culmination of 50 years of research. Could be the last harrah of the NIH with the amount of cuts we've had and the scientists who are taking jobs in other countries.

Unfortunately a staggering amount of research in other countries is largely funded by the NIH/USA.

How so?
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