logoalt Hacker News

forgotpwagainyesterday at 7:41 PM1 replyview on HN

A thread from yesterday about why gene therapy hasn't reached its potential: https://news.ycombinator.com/item?id=44573193


Replies

trhwayyesterday at 10:18 PM

Interesting 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.

show 2 replies