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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

#331

Post by raldi »

mondaygreens wrote: post #328
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.

How can they pass it on when they don't have the defect any more?

The DNA was only edited in the liver. But by the time this baby grows up and starts a family, we'll probably be able to fix that, too.

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

#332

Post by nahsra »

mondaygreens wrote: post #328
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.

How can they pass it on when they don't have the defect any more?

Gene editing is still pretty crude in terms of delivery.

Just because you can hit some germ-line cells in the liver, for example, doesn’t imply you’ll have good penetration into the reproductive organs.

We can’t zap people and change all their DNA at once, unless we can intervene at the point it’s just a few cells.

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

#333

Post by verisimi »

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…

Once the gene has been edited, things will work. But at some point that cell will die. Why would the replacement cell also have the edit? The DNA in the rest of the body's cells will still not be correct.

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

#334

Post by poilcn »

mondaygreens wrote: post #328
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.

How can they pass it on when they don't have the defect any more?

It only affects some cells, not the whole body.

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

#335

Post by Tade0 »

efilife wrote: post #297
squigz wrote: post #282

Earlier quoted context omitted.

I didn't say you did. I was trying to understand your point, and so was inferring what you could possibly have meant with your original comment.

Oh, sorry. I definitely think a father can (should?) celebrate medical advancements like this, and definitely shouldn't undermine death like "Ah, that's life". My point is that people often worry about their children's death when they themselves are still alive. Death seems okay if it's when they don't get to see it

Death of someone whose potential was largely realized is a very different thing than the death of someone who never got a chance at the same.

I would be deeply unhappy to learn that my children won't live to old age.

Also witnessing the death of a loved one is obviously traumatic. People grieve their parents dying of old age.

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

#336

Post by mmooss »

jordanpg wrote: post #124
declan_roberts wrote: post #101

Earlier quoted context omitted.

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

And so what?

Indeed - does it matter who performed the research? If the CRISPR reasearch were performed in another country, would that change the outcome for the infant?

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

#337

Post by esalman »

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…

Thanks to DOGE you might read less and less about this kind of things.

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

#338

Post by riffraff »

verisimi wrote: post #333
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…

Once the gene has been edited, things will work. But at some point that cell will die. Why would the replacement cell also have the edit? The DNA in the rest of the body's cells will still not be correct.

When cells duplicate they have the same (altered) DNA so the mutated cells survive.

You'll end up with mosaicism (cells with different DNA) but presumably you have enough of the new cells to fix the problem the original ones had.

You don't need to fix all the body, you just need to fix some of the, say, liver, and you're good.

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

#339

Post by kmmlng »

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…

I suppose in the beginning, it was about finding ways to measure how effective different altruistic approaches actually are and focusing your efforts on the most effective ones. Effective then essentially means how much impact you are achieving per dollar spent. One of the more convincing ways of doing this is looking at different charitable foundations and determining how much of each dollar you donate to them actually ends up being used to fix some problem and how much ends up being absorbed by the charitable foundation itself (salaries etc.) with nothing to show for it.

They might have lost the plot somewhere along the line, but the effective altruism movement had some good ideas.

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

#340

Post by agos »

autoexec wrote: post #9
colechristensen wrote: post #5

>KJ has made medical history. The baby, now 9 ½ months old, became the first patient of any age to have a custom gene-editing treatment, according to his doctors. This is _not_ the first human to be treated with a treatment under the wide umbrella of gene therapy based on their own edited genes. There probably is a more narrow first here but the technical details get lost in journalism which is a shame.

Okay, I'll bite: Who then was the first patient of any age to have a custom gene-editing treatment?

I remember six kids treated with a HIV retrovirus based therapy in 2013 in Italy
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