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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

#171

Post by monkeycantype »

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 remember from a few few years back that the lipid coating may have caused problems for the liver, when treating people for diseases that needed to target a lot of tissue, such as muscle disorders. Is that still the case?

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

#172

Post by sfink »

catigula wrote: post #159
sfink wrote: post #149

Earlier quoted context omitted.

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 no…

Sure, the purpose was to illustrate a slippery slope, and curing autism is meant to be more obviously good than abolishing all forms of neurodivergence but less obviously good than fixing fatal hereditary diseases.

I'm not going to claim that I know the perfect place to draw the line.

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

#173

Post by joshmarlow »

poyu wrote: post #31
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…

Made it sound like it's a computer, is it Turing complete?

If this thread interests you, you should check out "Blood Music" by Greg Bear. It's pretty old but the premise is that a researcher 'closes the loop' in a bunch of cells by making them able to edit their own DNA - thus making them Turing Complete.

Hilarity subsequently ensues.

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

#175

Post by jjeaff »

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…

I'm not an expert, but I have learned that FDA approval is not actually necessary for treatments and drugs. Your doctor has a lot of leeway when it comes to treatment but she of course experiences more risk of accusations of malpractice when prescribing off label drugs or unapproved treatments. insurance will also rarely cover treatment that is not FDA approved. the requirement for FDA approval generally has more to do with your legal ability to market the drug, treatment, or product.

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

#176

Post by sfink »

throw7 wrote: post #63

Gattaca here we come!

There is a strong hunger for Gattaca.

Heck, if parents could provide a trust fund for their kids in a way that their kids couldn't piss it away, they'd be all over it. (I'm sure this exists to varying degrees.)

Look at what wealthy parents already do to get their kids into colleges or out of jail. I think it's ridiculously naive to think that we parents wouldn't jump at the chance to write generational wealth into our kids' genes.

(This is not an argument that developing this capability is a bad thing and should be stopped.)

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

#177

Post by Muromec »

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.

That would be less effective than bio and chemical weapons are. Which are not used because they just suck

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

#178

Post by zmmmmm »

catigula wrote: post #159
sfink wrote: post #149

Earlier quoted context omitted.

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 no…

I think a better reason autism is a bad example is that part of its definition is that it is a consequence of fundamental brain structure and development (differentiating it from other psychological disorders which are acquired and more malleable). These aren't things you will "undo" with some gene edits. The whole brain has developed in a different way. Short of re-growing them a new brain you aren't going to change that (assuming you wanted to).

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

#179

Post by anthk »

koeng wrote: post #65
poyu wrote: post #31

Earlier quoted context omitted.

Made it sound like it's a computer, is it Turing complete?

It's fundamentally different than a computer and arguably more complete. The talk of "crawling along the genome" is kinda fundamentally wrong though and is a bit irking - CRISPR kinda just bumps around until it hits a PAM site, in which case it starts checking against sgRNA. Much more random than they make it seem

This is crazier: https://www.sciencealert.com/are-we-all-quantum-computers-wi...

About CRISP, it's like the ultimate Perl+Regex for the body.

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

#180

Post by dekhn »

joshmarlow wrote: post #173
poyu wrote: post #31

Earlier quoted context omitted.

Made it sound like it's a computer, is it Turing complete?

If this thread interests you, you should check out "Blood Music" by Greg Bear. It's pretty old but the premise is that a researcher 'closes the loop' in a bunch of cells by making them able to edit their own DNA - thus making them Turing Complete. Hilarity subsequently ensues.

Cells are already able to edit their own DNA. Examples include the yeast mating switch, in which the "active" gene is replaced by one of two templates, determining the role the yeast plays in mating (https://en.wikipedia.org/wiki/Mating_of_yeast#Mechanics_of_t...)

Further, your immune system does some clever combinatorial swapping to achieve diversity (https://en.wikipedia.org/wiki/V(D)J_recombination). The generated diversity is then screened by the immune system to find highly effective antibodies that bind to specific foreign invaders.

Doing something actually interesting from an engineering perspective makes for fun science fiction, but as always, the specific details in that story would be a very unlikely outcome.

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