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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

#91

Post by bilekas »

> 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 sometimes hard for regular people to appreciate just how much good actually comes out of Federal Funding. There was another thread where someone even said something along the lines of : "Well during war things get done faster" . This simply isn't true. It might be done louder but Federal Funding never stopped pushing things forward.

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

#92

Post by baxtr »

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…

Now imagine DOGE team of experts cutting this a couple of years ago

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

#93

Post by bilekas »

baxtr wrote: post #92
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…

Now imagine DOGE team of experts cutting this a couple of years ago

I didn't want to bring up specifics but I'd be lying if it wasn't on my mind.

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

#94

Post by pfisherman »

SquirrelOnFire wrote: post #44
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…

30 million people in the US are affected by "rare" genetic conditions.

Also rare genetic diseases give insight into the underlying mechanisms and pathology of common sporadic diseases, which can be leveraged to develop new and better therapies.

Getting a new drug or therapy approved for a rare form of a disease and then expanding the indication to the common disease patient population is a well established strategy.

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

#95

Post by Balgair »

Traubenfuchs wrote: post #72

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 coming from things like dirty water and bad food, but as we're solving those low hanging fruit, we have to go to lower probability events to make progress that feels equally important.

Also, if I am wrong here on the answers to the questions, please correct me!

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

#96

Post by pfisherman »

dekhn wrote: post #82
SquirrelOnFire wrote: post #44

Earlier quoted context omitted.

30 million people in the US are affected by "rare" genetic conditions.

Yes, but the cures here aren't general. They're highly specific, and the rare conditions have a long tail- large numbers of different conditions, each with a very small population of affected individuals, and likely, the treatments will be somewhat customized for each type of disease.

See my comment above. Getting approval for rare diseases and expanding the indication to the common form of the disease is a well established strategy in pharma.

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

#97

Post by shadowgovt »

bilekas wrote: post #93
baxtr wrote: post #92

Earlier quoted context omitted.

Now imagine DOGE team of experts cutting this a couple of years ago

I didn't want to bring up specifics but I'd be lying if it wasn't on my mind.

It would probably be good if more of us brought up specifics more often.

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

#98

Post by 0_____0 »

baxtr wrote: post #92
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…

Now imagine DOGE team of experts cutting this a couple of years ago

Here's the thing - likely few would have noticed. We are structurally blind to the places in which public investment would have made our lives better, especially when they are things like scientific research that the vast majority never think about until it produces results.

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

#99

Post by Teever »

Balgair wrote: post #83
MrZander wrote: post #11

> 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…

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 loathe things like vaccines and genuinely believe that vaccines can spread illness to the non-vaccinated feel the same way about other things like genetic modification and use legal mechanisms to discriminate and persecute people who are genetically modified.

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

#100

Post by anarticle »

vessenes wrote: post #40

NYT isn’t super specific here, but they made it sound like the disease treated is liver related. My understanding is that the liver is a good place to start with CRISPR-type gene treatments, in that the liver normally deals with anomalous shit in your bloodstream, say, like CRISPR type edits. So anywhere outside the liver is going to be significantly harder to get really broad uptake of gene edits. It’s crazy encoura…

Specifically it is this: https://en.wikipedia.org/wiki/Carbamoyl_phosphate_synthetase...

People born with this lack the enzyme CPS1, which screws up the urea cycle and causes a build up of ammonia. Ammonia build up is bad for your nervous system.

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