Scientists edited genes in a living person and saved his life | Hacker News Reader

Scientists edited genes in a living person and saved his life

popularmechanics.com

123 pointsQueensGambit44 comments

Paper: https://www.nejm.org/doi/full/10.1056/NEJMoa2504747

There was a guy on YouTube who genetically engineered himself to gain lactose tolerance. (https://www.youtube.com/watch?v=J3FcbFqSoQY)

It lasted for around a year and a half (https://www.youtube.com/watch?v=aoczYXJeMY4) before the effect mostly wore off. He took it orally, so it only affected his intestinal lining, and I presume it didn't effect enough stem cells to get a permanent effect, but it would still be usable as something taken annually, which is still far less often than any medication.

Wow this is amazing. Definitely the best biology video I've seen.

Edit: this is cool enough to deserve it’s own post, Im going to submit it: https://news.ycombinator.com/item?id=46405855

Agreed. If this isn't hacking then I don't know what is.

̶I̶ ̶t̶h̶i̶n̶k̶ ̶h̶e̶ ̶a̶c̶t̶u̶a̶l̶l̶y̶ ̶g̶e̶n̶e̶t̶i̶c̶a̶l̶l̶y̶ ̶e̶n̶g̶i̶n̶e̶e̶r̶e̶d̶ ̶h̶i̶s̶ ̶g̶u̶t̶ ̶b̶a̶c̶t̶e̶r̶i̶a̶ ̶r̶a̶t̶h̶e̶r̶ ̶t̶h̶a̶n̶ ̶a̶n̶y̶ ̶o̶f̶ ̶h̶i̶s̶ ̶o̶w̶n̶ ̶c̶e̶l̶l̶s̶ ̶t̶h̶e̶r̶e̶,̶ ̶r̶i̶g̶h̶t̶?̶

EDIT: Above is false. Went back and checked and I had mis-remembered the video.

From the video, it sounds like he engineered his own cells. Using a virus that is known for transferring genetic material into other organisms, he added a gene for producing lactase, and then ate it. I suppose that would affect both his gut bacteria and his own cells. But it lasted for ~1.5 years, which probably indicates that it truly was his cells. Also he seems to know what he's talking about, and he claims it was his own cells.

You're right, thanks!

Could be placebo though?

Is there a reason why I'm seeing squares between each of the characters in your message? It's making it pretty hard to read...

I'm using Chrome on Android

Firefox on iOS, I see strikethrough but the strikes are at varying heights.

The text is crossed out using Unicode combining characters of a strikethrough. This allows it to display without any specific formatting support, but it does require that the font support those characters. The font you're using doesn't support the characters, so it displays boxes, instead.

I thought (maybe mistakenly) that your body developed immunity to the adenovirus used to edit your own DNA, and so this trick could only be used once? Am I wrong?

In this specific instance the code he built used a non-human lactase-enzyme producing gene, which he states in the retrospective is very likely the reason why his immune cells started attacking. There was also the matter of some of the other coding pieces being non-ideal.

Your genes seem to be out of date, there are several security updates pending. The download will be 3.2 Gb and will take about 3 seconds..... Because this is a security update you can not refuse it. If you are commanding a vehicle park it now or switch to autopilot. Commencing update in 10, 9, 8, ...

the good old days when updates actually improved product quality :)

I updated to version 10.5.7 and now I see Samsung ads in my peripheral vision, wtf

You're the lucky one... they're a/b testing this and I get them flashing full size for a half second twice a day. I've tried blocking them at the firewall but their AI is so good now it can hack that in a couple of seconds, and they're using what I've seen as input to their bypass. It won't be long before we won't be able to turn it off at all. Joe Jackson saw it coming...

https://www.youtube.com/watch?v=GgqwdYAmoFQ

I am now nicotine addicted without smoking a single cigarette.

Very dumb question, but how exactly does gene therapy work in vivo? Don't you have to modify every cell (or affected type of cell) with a new copy of the DNA? How does that actually.... happen?

> Don't you have to modify every cell (or affected type of cell) with a new copy of the DNA?

It depends on the specific disorder that’s being treated. For some of them, targeting a fraction of the affected cells is enough to get the desired effect.

If the affected protein is actively causing problems, then you'd need to get most of the cells. But if it's just inert and not doing its job then you only need to get enough cells to get the production of the right version up to a somewhat functional level.

It's like trying to persuade enough people (cell) to change their mind so a social revolution takes place (cure). The argument (gene vector) must be very popular.

What worries me is the attacks on universities and the breaking of public private research based on this comment from the article:

"In a race against time, scientists and doctors across the U.S. developed the first in vivo gene therapy, thanks to decades of medical research."

With Trump it appears a window is closing on the development of important technologies and research. I doubt we will enter a new dark age, but in some areas, progress is likely to slow, which in turn will be used as evidence that it isn't worth funding, which will cause funding cuts and result in even slower progress. Everyone is racing to get stuff done, because there might not be the money tomorrow. It seems likely to me that longer term, more ambitious projects are probably being sidelined because there isn't time to complete them before the pencil pushers in the whitehouse defund them.

Look at how the Nixon administration and congress gutted NASA. It took nearly 40 years to crawl out of the hole in the roadmaps that their shortsighted stupidity created. We could have had reusable rockets, aerospike engines and Nuclear Thermal Propulsion in the early 1980s. Instead we got halfway measures like the space shuttle and the ISS that both ate budget but didn't create the required innovation for lower cost to orbit.

I couldn’t have said it better myself. They will use this to say not productive don’t fund.

My feeling is that rather than a snuffing out, we are witnessing a passing of the torch of the light of progress.

Better start learning mandarin.

Science works better with collaboration. If China and Europe have to take up the slack from a self-inflicted wound to US research, science as a whole suffers. There is far more science to do than scientists and when science funding gets canceled the number of scientist decrease.

After the US canceled the SSC, Europe built the LHC. The LHC isn't a one for one replacement of the SSC, but it would probably not have been built if the SSC had been built. What scientific projects would Europe have built instead? What did we lose?

>>I doubt we will enter a new dark age

But only to the extent that the regime fails to get its way.

If the regime continues, they will definitely push us back to a dark age, partially intentionally, partially through not caring about the future or being too stupid to figure out the consequences. And Theil, Musk, Vance and that whole lot are part of the crew pushing for that — technology for me but not for thee.

Unfortunately one of the common responses is "cancer is still deadly doesn't seem like they're doing anything but getting rich". These therapies aren't widely known and people are willing to throw them out for that reason.

The facts of life... to make an alteration in the evolvement of an organic life system is fatal. A coding sequence cannot be revised once it's been established.

Why not?

Because by the second day of incubation, any cells that have undergone reversion mutation give rise to revertant colonies, like rats leaving a sinking ship; then the ship... sinks.

What about EMS-3 recombination?

We've already tried it - ethyl, methane, sulfinate as an alkylating agent and potent mutagen; it created a virus so lethal the subject was dead before it even left the table.

Then a repressor protein, that would block the operating cells.

Wouldn't obstruct replication; but it does give rise to an error in replication, so that the newly formed DNA strand carries with it a mutation - and you've got a virus again... but this, all of this is academic.

You were made as well as we could make you.

Direct link to downloadable paper:

https://pmc.ncbi.nlm.nih.gov/articles/PMC12713542/

Story from May;

Discussion then: https://news.ycombinator.com/item?id=43997636

What enabled this treatment to be used now? Gene editing techniques have existed for a long time, but there were many reasons why they weren't being used in humans, like concerns about off-target edits and heritability. The article mentions something about gold nanoparticles, but this aspect was developed over the course of a few weeks, and in any case these aren't new either.

Well, CRISPR-Cas9 as a tool for genetic engineering was only invented in 2012. A 13-year translation timeline is not unusual, maybe even unusually fast. CAR-T cell therapy for cancer took 30 years from discovery to clinic. It took about 30 years to go from the early attempts to use engineered lipid nano particles for drug delivery to the first FDA-approved medication using them, doxil.

With CRISPR, it took a long time to figure out how to reliably edit just the gene you want and acceptably minimize off-target edits, including by delivering the therapeutic to just the organ affected and getting the dose and release right.

The public is understandably leery about experimental medical techniques. If they had killed this newborn child with CRISPR therapy, then it might have set created a backlash delaying translation of this technique for years, possibly decades.

In biomedicine, we’re always looking for therapies that approximate the level of precision control available in software. Unfortunately, it’s never more than an approximation, and our ability to measure and predict the size of that error is always limited. That is why the field moves slowly.

I mean to ask 'why now?' not 'what took so long?' What about the regulations or the science let this happen now, and not 5 years ago or 5 years into the future?

Also do we know that this was CRISPR?

Yes, CRISPR’s mentioned in the article. Someone should write a book about it to break down the history leading up to this accomplishment.

Yes, This was a CRISPER technique.

https://www.chop.edu/news/worlds-first-patient-treated-perso...

Expanding the timeline a bit, CRISPR was known as a possible gene targeting/editing tool by 2008 at least - I distinctly recall learning about it then in a guest lecture.

From the paper: "After regulatory approval had been obtained for the therapy, ..."

Are the documents relating to this FDA (?) approval application public? I'm curious about where the current boundaries lie and how the process works.

Ho does one just summon a group of highly skilled researchers to help out a baby? Like, how did these researchers get paid?

That's promising. Where do I sign up? I need my entire DNA code reprogrammed :)

https://pmc.ncbi.nlm.nih.gov/articles/PMC12713542/

(without paywall)

"Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease"

https://www.nejm.org/doi/full/10.1056/NEJMoa2504747

Note: from May 2025. https://gwern.net/doc/genetics/editing/2025-musunuru.pdf

Thanks! We've put this link in the header of the thread.

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