Scientists edited genes in a living person and saved his life
1–10 of 47 posts
Re: Scientists edited genes in a living person and saved his life
#2"Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease"
Re: Scientists edited genes in a living person and saved his life
#3From 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.
Re: Scientists edited genes in a living person and saved his life
#4"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
Re: Scientists edited genes in a living person and saved his life
#5What 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.
Re: Scientists edited genes in a living person and saved his life
#6There 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.
Re: Scientists edited genes in a living person and saved his life
#7What 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."
Re: Scientists edited genes in a living person and saved his life
#8What 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.
Re: Scientists edited genes in a living person and saved his life
#9"Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease" https://www.nejm.org/doi/full/10.1056/NEJMoa2504747
Thanks! We've put this link in the header of the thread.
Re: Scientists edited genes in a living person and saved his life
#10What 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 tim…
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?