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Baby is healed with first personalized gene-editing treatment

nytimes.com

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

#391

Post by tempaway43563 »

maxerickson wrote: post #208
Balgair wrote: post #83

Earlier quoted context omitted.

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…

Is this a troll? Pseudouridine mRNA isn't gene editing.

[dead]

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

#392

Post by ionwake »

Balgair wrote: post #255
maxerickson wrote: post #244

Earlier quoted context omitted.

I dunno, I think they are being sloppy and conflating things. We can induce manufacture of proteins and can design proteins that carry out gene editing, so we can stack that knowledge together to induce cells to manufacture proteins that carry out gene edits, but it's the payload that is the gene editing, not the instruction to make the protein. Given the merry movement to call the COVID vaccines gene editing, it ran…

Hey, yeah, I'm not the most up to date on the current methods. Most of my knowhow is a bit out of date here. So thanks for piping up to correct things. Do you know of any good resources that I can use to get up to speed on the exact methods they used for the baby? My understanding, outdated as it is, is that we're using the mRNA to go in and create CRISPR-CAS9 slicers/dicers and additionally to that, the correct gene…

I think you're replying to someone edgelord about covid who got confused about some mrna statement and then back pedalled re-affirming what the article was about.

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

#393

Post by tempaway43563 »

bilekas wrote: post #91

> But KJ’s treatment — which built on decades of federally funded research — offers a new path for companies to develop personalized treatments without going through years of expensive development and testing. Really incredible story and I'd love to know the process for receiving this, for example FDA approval etc. It's nice to see such in-your-face results from Federal funding programs. Without being political, it's…

they were able to develop the treatment and fast track it through the FDA in six months, details in this write-up

https://innovativegenomics.org/news/first-patient-treated-wi...

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

#394

Post by mushi01 »

morsecodist wrote: post #317
gr_norm wrote: post #243

Earlier quoted context omitted.

"Effective Altruism", something I find myself aligned with but not to the extremes taken by others.

Effective Altruism is such an interesting title. Almost no one views their Altruism as ineffective. The differentiator is what makes their flavor of Altruism effective, but that's not in the title. It would be like calling the movement "real Altruism" or "good Altruism". A good name might be rational Altruism because in practice these people are from the rationalist movement and doing Altruism, or what they feel is A…

Do you really think all altruism is effective? Caring about the immediate well-being of others is not as effective as thinking in the long term. The altruism you are describing is misguided altruism, which ultimately hurts more than it helps, while effective altruism goes beyond the surface-level help in ways that don't enable self-destructing behaviours or that don't perpetuate the problem.

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

#395

Post by something098 »

dekhn wrote: post #114
Balgair wrote: post #95

Earlier quoted context omitted.

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…

Both eggs and sperm producing cells are created during formation of embrio. We could even define that these cells as "clones of you" that were created not much after your first cells were created, because your DNA, that undergoes changes in your organism is never given to your offsprings - it is DNA of your "clone" cells. Eggs are as you have defined - "very old cells". Most probably cells that are producing sperm also can be defined as "very old cells" as most probably sperm production does not function the same way as other cells, that are staying in organism and accumulate mutations.

Stem cells from other organs has absolutelly nothing to do with this. Unless you are refering to procedures of planting stem cells from one organ to another to help failing organ, as stem cells are universal cells, that are able to produce cells for any organ.

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

#397

Post by palisade »

rkangel wrote: post #361
palisade wrote: post #322

Does this mean when they grow up, their own offspring will also have this defect and require a correction? And, if so, does this mean it is now introducing this defective gene into our gene pool? I know this is an issue with caesarean section. It is becoming more prevalent because those who require it are surviving, making it more likely to happen in their offspring.

> know this is an issue with caesarean section. It is becoming more prevalent because those who require it are surviving You state this as a fact and I've heard it as a strong hypothesis, but I wasn't aware of much evidence to confirm it?

"The cesarean delivery rate increased from 5% in 1970 to 31.9% in 2016. This sharp increase can be attributed to various factors, including changes in maternal age, medical advancements allowing more complicated pregnancies to proceed, and evolving obstetric practices. In 2022, the United States recorded more than 3.66 million births, most of which resulted from spontaneous or induced labor. Labor dystocia remains the most common indication for primary cesarean delivery. Globally, cesarean delivery rates continue to rise, and reducing unnecessary cesarean procedures remains a priority in the United States, where 32.2% of all births in 2022 were cesarean deliveries."

https://www.ncbi.nlm.nih.gov/books/NBK546707/

"If this trend continues, by 2030 the highest rates are likely to be in Eastern Asia (63%), Latin America and the Caribbean (54%), Western Asia (50%), Northern Africa (48%) Southern Europe (47%) and Australia and New Zealand (45%), the research suggests."

https://www.who.int/news/item/16-06-2021-caesarean-section-r...

Note: Coincidentally, WHO's article I've linked is lamenting that Sub-saharan Africa only had 5% cesarean due to less availability of the procedure. It is their perspective that the increase in percentages is a good thing and indicates progress, instead of being concerning. And, they find Sub-saharan Africa's low numbers concerning, instead.

Side Note: I also found lots of interesting articles which I haven't posted here, about epigenetic side effects caused by caesarean deliveries like leukemia, illnesses and other genetic issues. But, that seems out of scope for your question. You can make a quick search and find these, though.

"A female-to-female familial predisposition to caesarean section was observed. It could be caused by biologic inheritance, primarily working through maternal alleles and/or environmental factors. The results imply that both mechanisms could be important."

https://pubmed.ncbi.nlm.nih.gov/18540028/

"Large-scale epidemiological studies indeed evidence that women born by C-section are more likely to deliver by Caesarean than women born vaginally, owing primarily to genetic rather than social factors."

https://www.pnas.org/doi/10.1073/pnas.1712203114

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

#398

Post by rkangel »

palisade wrote: post #397
rkangel wrote: post #361

Earlier quoted context omitted.

> know this is an issue with caesarean section. It is becoming more prevalent because those who require it are surviving You state this as a fact and I've heard it as a strong hypothesis, but I wasn't aware of much evidence to confirm it?

"The cesarean delivery rate increased from 5% in 1970 to 31.9% in 2016. This sharp increase can be attributed to various factors, including changes in maternal age, medical advancements allowing more complicated pregnancies to proceed, and evolving obstetric practices. In 2022, the United States recorded more than 3.66 million births, most of which resulted from spontaneous or induced labor. Labor dystocia remains th…

Interesting - thank you!

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

#399

Post by flakeoil »

fendy3002 wrote: post #259
tuna-piano wrote: post #224

If someone in the year 2050 was to pick out the most important news article from 2025, I won't be surprised if they choose this one. For those who don't understand this stuff - we are now capable of editing some of a body's DNA in ways that predictably change their attributes. The baby's liver now has different (and better) DNA than the rest of its body. We still are struggling in most cases with how to deliver the D…

the usual next questions will be: - how further can we push this to make the best, most optimized human? - what are moral implication of this? - what are the side effects / downsides?

I also wonder what happens if this kid one day has kids. In this case it was a very rare genetic disease, but if the same was applied to a less rare genetic disease (where it is also more beneficial to have a treatment as more people have use of it) wouldn't the end result be that more and more kids will be born with these diseases?
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