[HN Gopher] Scientists edited genes in a living person and saved...
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Scientists edited genes in a living person and saved his life
Paper: https://www.nejm.org/doi/full/10.1056/NEJMoa2504747
Author : QueensGambit
Score : 84 points
Date : 2025-12-27 18:26 UTC (4 hours ago)
(HTM) web link (www.popularmechanics.com)
(TXT) w3m dump (www.popularmechanics.com)
| gavinray wrote:
| _" Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic
| Disease"_
|
| https://www.nejm.org/doi/full/10.1056/NEJMoa2504747
| gwern wrote:
| Note: from May 2025.
| https://gwern.net/doc/genetics/editing/2025-musunuru.pdf
| tomhow wrote:
| Thanks! We've put this link in the header of the thread.
| lysace wrote:
| 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.
| qnleigh wrote:
| 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.
| directevolve wrote:
| 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.
| qnleigh wrote:
| 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?
| directevolve wrote:
| Yes, CRISPR's mentioned in the article. Someone should
| write a book about it to break down the history leading up
| to this accomplishment.
| rolph wrote:
| Yes, This was a CRISPER technique.
|
| https://www.chop.edu/news/worlds-first-patient-treated-
| perso...
| hirsin wrote:
| 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.
| dlcarrier wrote:
| 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.
| pinkmuffinere wrote:
| 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
| c1ccccc1 wrote:
| I think he actually genetically engineered his gut bacteria
| rather than any of his own cells there, right?
| pinkmuffinere wrote:
| 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.
| thrance wrote:
| 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?
| gigatexal wrote:
| 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."
| EthanHeilman wrote:
| 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.
| gigatexal wrote:
| I couldn't have said it better myself. They will use this to
| say not productive don't fund.
| nyc_data_geek1 wrote:
| 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.
| toss1 wrote:
| >>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.
| jacquesm wrote:
| 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, ...
| bflesch wrote:
| the good old days when updates actually improved product
| quality :)
| msla wrote:
| Direct link to downloadable paper:
|
| https://pmc.ncbi.nlm.nih.gov/articles/PMC12713542/
| probe wrote:
| 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?
| ChrisArchitect wrote:
| Story from May;
|
| Discussion then: https://news.ycombinator.com/item?id=43997636
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