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.
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.
Your genes seem to be out of date, there are several security updates pending.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, ...
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?
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.
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[ 3.2 ms ] story [ 50.1 ms ] threadhttps://www.nejm.org/doi/full/10.1056/NEJMoa2504747
Are the documents relating to this FDA (?) approval application public? I'm curious about where the current boundaries lie and how the process works.
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.
"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."
https://pmc.ncbi.nlm.nih.gov/articles/PMC12713542/
Discussion then: https://news.ycombinator.com/item?id=43997636
(without paywall)
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.