100 points giuliomagnifico 3 days ago 17 comments
raomin 13 hours ago | parent
divy77 12 hours ago | parent
fnordpiglet 12 hours ago | parent
bitwize 11 hours ago | parent
wetwater 11 hours ago | parent
Jokes apart, can anyone well versed actually tell of this is just a paper for publishing sake or is there any truth to this story ?
refactor_master 10 hours ago | parent
“In mice” - actually not even.
vlovich123 8 hours ago | parent
ungreased0675 10 hours ago | parent
I do hope this is serious research as well. It’s hard to tell from the outside.
adriand 9 hours ago | parent
That’s so cool. It reminds me of being a kid and playing with magnets beneath a sheet of cardboard, using them to invisibly move metal objects on the surface. Is that sort of the principle here?
Konnstann 7 hours ago | parent
stalfie 2 hours ago | parent
The main issue is delivering the genetic patch to the tissue you want to fix. Some genes (eg. BRCA) are just really big and can't fit on some vectors, some tissues (eg. brain) are hard to reach, and you want to try to target tissues selectively, since we're not sure yet about side effects like insertional mutagenesis (although it doesn't seem to be a huge problem atm...probably depends on on the vector). A good vector makes a huge difference. Lipid nanoparticles for instance accumulate in the liver and can deliver all kinds of stuff, hence why there are so many studies targeting genetic errors of liver metabolism.
Exotic vectors for normal drugs are usually useless, as it would be too expensive for stuff needing daily or even monthly dosing (as long as it doesn't have a property of selectively targeting cancer). But for single dosage therapies, which gene therapies are, they are a potentially essential tool in the toolbox.
Edit: after writing this comment I just happened to check NEJM, and boom, yet another lipid nanoparticle based gene therapy in phase 3 which tremendous results: https://www.nejm.org/doi/full/10.1056/NEJMoa2600931?query=fe...
Linked this to illustrate the point. This is also a good example because the targeted genetic "patch" doesn't actually fix the underlying problem directly (C1 inhibitor deficiency), but goes about fixing the problem in a roundabout way just because that particular way involves the liver, instead of having to deliver the patch to all affected tissues (which in this disease is pretty much all of them). The treatment is thus just hugely effective instead of a complete cure. I personally have dealt with difficult cases of this particular disease in the past, and this treatment is a huge deal.
alexpotato 34 minutes ago | parent
The doctor that invented the "blood vessel path to the heart for a catheter" was German. He had the idea to try it out, grabbed a regular catheter, inserted it into a blood vessel in his own arm and pushed it in till he felt it in his heart.
He wanted evidence that he had actually done this so he walked to the X-Ray department (he worked at a hospital) and asked them to get him a chest x-ray.
Wild how medicine has progressed.
(This story is from The Body by Bill Bryson)