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New Gene Therapy technique used for first time to correct a gene in live animal

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K

Kinitari

Black Canada Mafia
http://phys.org/news/2014-03-reverse-liver-disorder-mice-mutated.html
Using a new gene-editing system based on bacterial proteins, MIT researchers have cured mice of a rare liver disorder caused by a single genetic mutation.
The findings, described in the March 30 issue of Nature Biotechnology, offer the first evidence that this gene-editing technique, known as CRISPR, can reverse disease symptoms in living animals. CRISPR, which offers an easy way to snip out mutated DNA and replace it with the correct sequence, holds potential for treating many genetic disorders, according to the research team.
"What's exciting about this approach is that we can actually correct a defective gene in a living adult animal," says Daniel Anderson, the Samuel A. Goldblith Associate Professor of Chemical Engineering at MIT, a member of the Koch Institute for Integrative Cancer Research, and the senior author of the paper.
The recently developed CRISPR system relies on cellular machinery that bacteria use to defend themselves from viral infection. Researchers have copied this cellular system to create gene-editing complexes that include a DNA-cutting enzyme called Cas9 bound to a short RNA guide strand that is programmed to bind to a specific genome sequence, telling Cas9 where to make its cut.
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Using this approach, the correct gene was inserted in about one of every 250 hepatocytes—the cells that make up most of the liver. Over the next 30 days, those healthy cells began to proliferate and replace diseased liver cells, eventually accounting for about one-third of all hepatocytes. This was enough to cure the disease, allowing the mice to survive after being taken off the NCTB drug.
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I feel like we're gonna do great things with gene therapy soon!
 
E

EdibleKnife

Member
61142-Mmmmm-science-Pepsi-can-gif-cDA2.gif
 
Rest

Rest

All these years later I still chuckle at what a fucking moron that guy is.
Very interesting. Medicine is getting better all the time.
 
B

Bulbo Urethral Baggins

Banned
Helacious said:
Please tell me this is gonna fix my psoriasis
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Definitely possible if it's gene related and I think psoriasis has been shown to be linked with a mutated gene. I think it's not that far off. Crispr is really an amazing system. It's already being used to knock out genes to make knockout animals. But more importantly, it can specifically edit or repair targeted mutant genes. Hell, it could also be used to create designer babies. This isn't some far away thing either. It's pretty new, but already a lot of progress is being made. It works in human cells and now it's been shown to work in a whole organism.
This is the real deal.
 
O

OldKentuckyShark

Member
Stinkles said:
Oh yeah, let's fix your trivial bullshit, meanwhile people are still bald.
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Not sure if the irony was intentional.


I'm concerned about the possibility of this developing horrible mutations that turn us into monsters, like in I Am Legend.
We'll also have to deal with mass protests from religious people and the like who believe such a thing is wrong.
 
davepoobond

davepoobond

you can't put a price on sparks
OldKentuckyShark said:
Not sure if the irony was intentional.


I'm concerned about the possibility of this developing horrible mutations that turn us into monsters, like in I Am Legend.
We'll also have to deal with mass protests from religious people and the like who believe such a thing is wrong.
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That's only a worry if those mutations are passed on.

But anything undesirable could just be geneticized out, so not sure why you're really worried
 
P

Platy

Member
Viewt said:
Hah! No kidding. "All righty there, Vincent. We fixed your heart, made you a bit taller, and corrected that myopia. Have fun in space!"
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I don't think gene therapy would make you taller ... but then again he only needed to be taller to pass as the other dude, which he would not need if this existed
 
R

Raist

Banned
Yeah, I'm using it (CRISPR). It's not as easy as it seems on paper, but it's quite powerful and the latest revolution for molecular biology tools. The potential is amazing.
I'm expecting it to be worth a Nobel prize down the line. It's quite fascinating how it was developed, too. Mostly serendipity.

Helacious said:
Please tell me this is gonna fix my psoriasis
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Unfortunately, psoriasis is a complex disease. Just like other forms of gene therapy, this is mostly applicable to monogenic diseases.
While CRISPR does have a simple and efficient potential for multiplexing, the problem with diseases like that is that it's not entirely clear which genes are involved, and there are many other factors involved. For things like psoriasis, the best bet is immunotherapy.
 
D

Drazgul

Member
Raist said:
Yeah, I'm using it (CRISPR). It's not as easy as it seems on paper, but it's quite powerful and the latest revolution for molecular biology tools. The potential is amazing.
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What are you working on, if you don't mind me asking?
 
R

raindoc

Member
Raist said:
Yeah, I'm using it (CRISPR). It's not as easy as it seems on paper, but it's quite powerful and the latest revolution for molecular biology tools. The potential is amazing.
I'm expecting it to be worth a Nobel prize down the line. It's quite fascinating how it was developed, too. Mostly serendipity.



Unfortunately, psoriasis is a complex disease. Just like other forms of gene therapy, this is mostly applicable to monogenic diseases.
While CRISPR does have a simple and efficient potential for multiplexing, the problem with diseases like that is that it's not entirely clear which genes are involved, and there are many other factors involved. For things like psoriasis, the best bet is immunotherapy.
Click to expand...

could it be used to repair tumor suppressor genes (p53, pRB)?
 
R

Raist

Banned
Drazgul said:
What are you working on, if you don't mind me asking?
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Not an actual disease, I'm a molecular immunologist. Well I guess my research area has potential implications for cancer immunotherapy, but not so directly anyway.
I'm just using CRISPR as a tool to study gene function. It really puts to shame all the other tools we had so far and the applications are ridiculous, especially for pure fundamental research. It's simple, cheap and extremely versatile. On the clinical side, it's also (presumably) a lot safer than other tools that have been used so far for gene therapy.

raindoc said:
could it be used to repair tumor suppressor genes (p53, pRB)?
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Yep. It can be used to repair any gene that has a detrimental mutation. That's just one of many applications though.
 
Monocle

Monocle

Member
Raist said:
Not an actual disease, I'm a molecular immunologist. Well I guess my research area has potential implications for cancer immunotherapy, but not so directly anyway.
I'm just using CRISPR as a tool to study gene function. It really puts to shame all the other tools we had so far and the applications are ridiculous, especially for pure fundamental research. It's simple, cheap and extremely versatile. On the clinical side, it's also (presumably) a lot safer than other tools that have been used so far for gene therapy.



Yep. It can be used to repair any gene that has a detrimental mutation. That's just one of many applications though.
Click to expand...
Really exciting stuff! Can't wait to see what this leads to over the next decade or so.
 
R

Raist

Banned
Monocle said:
Really exciting stuff! Can't wait to see what this leads to over the next decade or so.
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Yep. The benefits of this are happening right now for fundamental research, but it will take a while for clinical stuff, as usual. The main problem being delivery but that's nothing new. I'm not too familiar with that field though, so I don't know what's hot right now.
 
B

Broken Joystick

At least you can talk. Who are you?
Maddocks said:
LD5lW3Xl.jpg

Soon.
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The next-generation Genome Soldiers who had undergone gene therapy began to exhibit a curious disorder in which their bodies became bilaterally symmetrical - a common trait in extinct species. Because in some cases, this led to sudden and unexplained death, certain units virtually collapsed.

Normally, animals' bodies have an asymmetrical structure suggesting that there was a mutation in the gene that determines asymmetry. The phenomenon was thought to be a side effect of the gene therapy.
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I'll pass.
 
M

Makai

Member
Will this go commercial in my lifetime? Colorblindness was cured in monkeys years ago and still no cure for humans.
 
Phoenix

Phoenix

Member
For the love of God, please keep the patent trolls away from this stuff. We need some form of legislation to prevent that level of interference with the progress of these developments in the House right now - instead of coming up with new ways to try to dismantle Obamacare.
 
K

kirby_fox

Banned
This could also cute a bunch of diseases like HIV/AIDS in theory. People who are immune to the disease have mutated DNA themselves, so it'd be interesting to see if taking their mutated DNA and splicing it into a person who isn't immune with the disease would make them immune and basically healthy again.

This could cure a bunch of diseases with no cure if someone is immune.
 
R

Raist

Banned
Phoenix said:
For the love of God, please keep the patent trolls away from this stuff. We need some form of legislation to prevent that level of interference with the progress of these developments in the House right now - instead of coming up with new ways to try to dismantle Obamacare.
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The method itself can't be patented - it's been entirely developed and published by research labs, and made widely available to the community. The only thing pharmas can do is develop and sell their own variant, but they can't get ownership of the basic principle.

Even biotech companies are a bit screwed on that one. They sell you "ready-made" stuff, but it's so easy to design that unless you have shitload of money and don't want to bother making some quick tests, you don't need them. All it takes is a few hundred dollars at worst, and a couple of weeks, and you can efficiently use this system. Of course, the commercialization of that thing is a different story.

kirby_fox said:
This could also cute a bunch of diseases like HIV/AIDS in theory. People who are immune to the disease have mutated DNA themselves, so it'd be interesting to see if taking their mutated DNA and splicing it into a person who isn't immune with the disease would make them immune and basically healthy again.

This could cure a bunch of diseases with no cure if someone is immune.
Click to expand...

There's a much easier and efficient way with this, potentially. The problem with HIV is while the virus itself can be kept reasonable in check now, it can't be eliminated because of its ability to stay dormant via its integration in the genome. This system could potentially allow to get rid of the HIV genome (or at least, fuck it up to a point that it's not functional anymore). Of course once again the problem is delivery.
 
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