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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

#151

Post by epistasis »

jordanpg wrote: post #122
javiramos wrote: post #55

Research funded by the NIH which our government is actively gutting

As the father of a 5 year old boy with a genetic degenerative muscular disease whose lifespan will depend directly on how fast these technologies progress, I have difficulty responding in a civilized manner to the pointless, cruel, and stupid actions of the Administration in this regard. Rage is the word. It is breathtaking to consider how the members of the Administration and their children, parents, and grandparent…

I have the same rage. But it extends equally to those who voted them in and donated to their campaigns, including my own family members.

They have created a huge rift in this country and I am still trying to figure out if I will forgive my family members and what they'd have to do to set us on a path towards reconciliation.

When there's a contract in place to conduct pediatric cancer research, and the government decides one day to break that contract, and it takes courts to rectify the situation, and then the government defies the courts, and the voters are cheering on the illegal actions of the politicians, well, rage is a mild word for what I feel.

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

#152

Post by Balgair »

alecco wrote: post #113
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…

> [...] 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.

Yeah, it's not a drama.

The reason that the body doesn't alarm as much to Pseudouridine, is that it's not a 'natural' RNA base. Meaning that, for the most part, nature really never uses it and so we haven't evolved to look out for it. Nature uses Uridine and so immune systems have evolved to look out for random bits of RNA in the body that use it and then clean that all up.

It's like if you're looking to clean up legos in you house with a romba that only cleans up legos. And all of a sudden it finds a duplo. It's going to take a hot second to figure out what to do with the duplo. And in that time, you can sneak by and build a duplo fort. (Look, I know this analogy is bad, but it's the best I can come up with on the fly, sorry. If anyone else wnats to come up with a better one, please do!).

The Pseudouridine is used up and degraded very quickly inside the cell, minutes at the very very longest, more like microseconds. It's just part of a messenger (the 'm' in 'mRNA') to tell the cell to do things.

You might see mRNA gene editing in factory farms, but it would just be easier to do germline editing instead and skip spraying animals, plants, and fungi. Why waste the equipment, right?

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

#153

Post by GenshoTikamura »

throw7 wrote: post #63

Gattaca here we come!

One can not simply raise valid concerns about gene editing technologies in the hands of the entities that don't hesitate sending people en masse to kill and die and otherwise manifest their fascist cravings in the open, here on HN and walk away undownvoted

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

#154

Post by XorNot »

rcpt wrote: post #39
pacoWebConsult wrote: post #13

Earlier quoted context omitted.

From a purely utilitarian perspective, funding research like this is not an effective use of dollars at the margin. How many people could we save if an equivalent amount was put into reducing obesity, smoking, and drinking? How many people could we save if we stopped spending money we don't have to do things that the government isn't competent at allocating anyways? That's not to say the research itself is not impres…

A huge amount of money went into researching anti obesity medications

Which it's worth noting, succeeded so wildly that 1/5th of Denmark's jobs growth last year was related to Ozempic production.

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

#155

Post by abracadaniel »

alecco wrote: post #113
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…

> [...] 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.

As I understand it, there is nothing in nature that can create it, so the mRNA can never be accidentally replicated. It’s a safety mechanism that prevents escape.

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

#156

Post by blangk »

pavlov wrote: post #150
ascorbic wrote: post #146

Earlier quoted context omitted.

A rare case where the list of awards she's received is so long it needs a separate Wikipedia page https://en.wikipedia.org/wiki/List_of_awards_and_honors_rece...

[flagged]

This seems like a non sequitur.

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

#158

Post by Balgair »

imcritic wrote: post #144
alecco wrote: post #113

Earlier quoted context omitted.

> [...] 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.

Farming? This will be used in warfare.

Not under the current way we do things, I don't imagine.

So the real trick here isn't the mRNA, it's the nanobubbles. Basically, you're putting these bits of mRNA into these little fat bubbles and then injecting those into the blood. Making those bubble shelf stable is really hard, hence the issues with temperature and the covid vaccine. To then make those little fat bubbles stable-ish in the blood is also a really hard thing to do. They have to get to the right places (in this baby's case, the liver) and then degrade there, drop off the mRNA, and not mess up other tissues all that much. Like, it's not terrible to make these micelles degrade in vivo, but to have them do that and not degrade in the tubes, ... wow... that is really difficult. There's a reason that Moderna is so highly valued, and it's these bubbles.

To try to then put these in a weapon that could do this though the airways would be, like, nearly impossible. Like, as in I think the second law of thermodynamics, let alone biology, and then simple industrial countermeasure like a N95 respirator, yeah, I think all of that makes it pretty much impossible to weaponize.

(Hedging my bets here: I don't know if they had to do all that with this baby, as you can kinda go from lab to baby really fast, since it's such a special case. But for mRNA based vaccines and cancer treatments, you have to deal with the shelf stable issue)

(Also, other bio people, yes, I am trying to explain to laymen here. Please chime in and tell me how I'm wrong here)

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

#159

Post by catigula »

sfink wrote: post #149
Teever wrote: post #99

Earlier quoted context omitted.

I suppose a downside (depending on your perspective) of this is that it will make people who are genetically modified in this fashion trivial to detect. That's good if your goals are to detect genetic modification which may be considered cheating in competitive sports. That's bad if your goals are to detect genetically modified people and discriminate against them. I see a near future where the kind of people who loa…

Careful with qualifiers there. I genuinely believe that vaccines can spread illness to the non-vaccinated, since it has happened many times and is well-documented. For example, it's why only the inactivated (aka "dead" virus) polio vaccine has been used in the US since 2000. I'm not arguing about whether the risks of the attenuated virus outweigh the benefits. I think the data are very clear there. (Heh -- and I'm su…

I mean, I feel like autism is a terrible example here, it's not just some quirky personality trait, it's a reality people live with that runs the gamut from difficult to completely debilitating. Even the more mild forms of autism cause extreme difficulty in many aspects of life. If that was curable or preventable, that'd be great.

If it turns out some pathogen or chemical made me autistic, regardless of whether or not I could be cured as an adult, I'd have certainly preferred to live the reality where I had been as a child.

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

#160

Post by primax »

WorkerBee28474 wrote: post #59
os2warpman wrote: post #41

Earlier quoted context omitted.

I think you may be operating under the assumption that the extremely expensive price tag will need to be repeated for each patient. In reality, as this process becomes more mature it is going to become inexpensive. The reduction in cost will almost certainly be similar to reduction in cost needed to sequence an individual's genome, which has fallen from tens of millions to hundreds of dollars. The only catch is that…

> In reality, as this process becomes more mature it is going to become inexpensive. There's no evidence to support that gene therapy will ever be inexpensive. We can merely say that the process may become less shockingly expensive.

> There's no evidence to support that gene therapy will ever be inexpensive. We can merely say that the process may become less shockingly expensive.

A similar thing has been said about so many cutting edge therapies and technologies in the past that I think you'll end up being quite surprised.

Eventually someone will invent a machine that spits these therapies out like espresso machines.

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