> I think if this worked as intended, no additional treatment is required.
It is correct if the therapy is targeting all stem cells. I have not understood clearly from the article but it seems the treatement is targeting "living cells". Once all your 'fixed' cells have been renewed you would need another round of treatement.
I have Haemophilia A, I was actually offered a place on an earlier trial although I didn't do it. It was explained to me that you would not need further gene treatments even though it targeted adult cells.
I think most cells are made by mitosis and don't come from stem cells.
Thanks for sharing! Can I ask what considerations went into your decision? Will you be pursuing this now that there is more evidence that it's effective?
I mean, obviously I regret it now, but at the time I had no idea it would work. I'm sure the next trial will be extremely oversubscribed! I think my trial was actually for a low dose version to test safety of the vector and it didn't have any clinical effects.
Its quite incredible though, there's only a few thousand people in the UK with haemophilia - this trial just cured like 0.5% of them
Would you mind keeping us updated if you pursue the next clinical trial? Happy to kick in Patreon money for your time. (Not a wierdo, just deeply interested in Curing All The Things).
well, stem cells are special in that they form tree replication structures to avoid problems like the Hayflick limit, so if the ones high up in the tree got repaired, you should have new stem cells born that inherit the fix for decades. At least, that's my understanding of the theory; that's no guarantee it works that way in reality.
It is correct if the therapy is targeting all stem cells. I have not understood clearly from the article but it seems the treatement is targeting "living cells". Once all your 'fixed' cells have been renewed you would need another round of treatement.