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.
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.
"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
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.
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.