First in vivo gene editing in humans in clinical trials

So not sz related. But a major scientific step in gene editing cures for various disease in humans.

https://www.google.com/amp/www.the-scientist.com/%3Farticles.amp/articleNo/49456/title/First-In-Vivo-Human-Genome-Editing-to-Be-Tested-in-New-Clinical-Trial/

4 Likes

Thanks for sharing. …twinkle…!!!

1 Like

This is really freaking exciting! I wish I was born earlier but in 20 or so years I will be old and likely suffering from aging related illnesses. If somewhere before then they discover how to treat such diseases with gene editing my life will be better. Truly a great article, and such a specific target disease.

Not sure if this will yield any progress in curing sz. Sz is cause by diffuse mutations in many genes unlike hemophilia. Additionally the brain is very poorly understood, as in we haven’t yet figured out exactly what goes wrong in sz. To make matters worse it seems sz could be a cluster of symptoms rather than a disease, or indeed as syndrome composed of several clusters of symptoms. We just know so little with sz, it is unlikely that gene editing will cure it.

Anyhow, thank you twinklestars for posting this article, I feel like this is “one small step”.

2 Likes

Yeah, I don’t know how likely gene editing is as a cure for sz. Probably theoretically possible though, since many gene variants are known. The main issue would probably be reintroducing them into the brain, and whether the expression of that gene was effecting current brain function and not only past brain development. Since some genes continue to effect levels of certain proteins that’s plausible. Possibly more plausible combined with stem cell transplants.

I read 3 or 4 articles on that today but it’s Parkinsons, Alzheimer’s, Huntingtons and ALS where the main focus is. That’s OK, they can work out the kinks there.

I did come across one article on both gene editing and stem cells in the treatment of arthritis. That’s still in mice though. https://www.sciencedaily.com/releases/2017/04/170427141703.htm

1 Like

Hi guys…when is it turn on sz…

1 Like

They need to find out the genetic underpinnings of negative symptoms. From there it will be a walk in the park.
(Wishful thinking)

1 Like

I have SZA, apparently. My therapist said my lack of motivation and drive is not from SZ but from major depression. I don’t know. I always blamed the SZ.

1 Like

Cogentin seems to improve my motivation but makes it harder to learn things.

I had a cat named cogentin once. She was really neat! Used to hang around and drink milk from a saucer outback of randy Watson general store

1 Like

sup dr green thumb …!!!

What about the zinc fingers?

No idea what those are but they definitely have comedy potential.