alt Hacker NewsInteresting point there:
"The other problem is with viral vector based gene therapy is you can’t have it again. You develop antibodies which prevent it from working again, and it could cause a dangerous immune response."
Just wondering - would it make sense to immune-suppress the patient for a short period of administering of the viral-based therapy.
And as they describe that most gene therapies affect only extra-nuclear DNA, and thus have no permanent effect, wouldn't mRNA work better then in such cases - naturally the tech wasn't there 10+ years ago, yet today thanks to COVID it is here.
Edit (due to posting rate limit) in response to comment below:
I was thinking about mRNA coding dystrophin like it was coding COVID protein - should be cheap and easy (well, for some definition of easy in that context) doable, and it would be like a weekly self-injection - no toxicity, etc. Of course fixing the issue once for life would be better, once such cure becomes available, yet for now it would be similar like diabetics have with insulin - hassle for sure, yet it works.
Replies
mRNA vaccines like the Pfizer and Moderna COVID vaccines don't enter the nucleus nor have a permanent effect. The mRNA breaks down after a few days.
AAV based therapies may have no permanent effect when the cells in question are actively proliferating (and the payload dilutes with each division) but muscle tissue is largely post-mitotic.
mRNA is in comparison very transient (in the range of days, and that's being charitable), even when modified (5' cap, uridine analogs, poly(A) tail) as it was in COVID vaccines. This is fine for vaccines, as you essentially want just a single exposure to the protein with each vaccine dose. You do need dystrophin continuously though (even though the cells are not dividing much, they are still recycling it).
You could argue for delivering gene therapy with mRNA/NLPs in multiple doses over the course of patient's life but that would likely 1) exacerbate toxicity and 2) be super-expensive