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.
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.
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.
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?
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.
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.
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.
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.
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, This was a CRISPER technique.
https://www.chop.edu/news/worlds-first-patient-treated-perso...
Yes, CRISPR’s mentioned in the article. Someone should write a book about it to break down the history leading up to this accomplishment.
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.
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.
"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.