US scientists try 1st gene editing in the body

Fingers crossed. I hope this works so it can help the many people who are suffering with diseases that are hard to treat.

Brian Madeux who is suffering with hunter syndrome received billions of copies of corrective genes and a genetic tool to cut the DNA in a precise spot.

OAKLAND, Calif. (AP) — Scientists for the first time have tried editing a gene inside the body in a bold attempt to permanently change a person’s DNA to cure a disease.

The experiment was done Monday in California on 44-year-old Brian Madeux. Through an IV, he received billions of copies of a corrective gene and a genetic tool to cut his DNA in a precise spot.

apnews.com...:-US-scientists-try-1st-gene-editing-in-the-body

In a month the scientists will see if it is working.

Signs of whether it’s working may come in a month; tests will show for sure in three months.

If it’s successful, it could give a major boost to the fledgling field of gene therapy . Scientists have edited people’s genes before, altering cells in the lab that are then returned to patients. There also are gene therapies that don’t involve editing DNA.

Very risky. Cutting your DNA open, inserting a gene and "stitching" it back up is very dangerous. Once it's done there is no going back, toying with mother nature is very very risky. The scientists don't know what the risks are. I am surprised that these tests are even being done in the US. FDA and all.

“We cut your DNA, open it up, insert a gene, stitch it back up. Invisible mending,” said Dr. Sandy Macrae, president of Sangamo Therapeutics, the California company testing this for two metabolic diseases and hemophilia. “It becomes part of your DNA and is there for the rest of your life.”

That also means there’s no going back, no way to erase any mistakes the editing might cause.

“You’re really toying with Mother Nature” and the risks can’t be fully known, but the studies should move forward because these are incurable diseases, said one independent expert, Dr. Eric Topol of the Scripps Translational Science Institute in San Diego.

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Well, maybe they will find out about the unknown consequences of their action by finding human test animals to mess up and study. They still do not know enough about companion genes working in sincro with other genes to figure out if the changes will not cause very bad results. Everyone is different, one size shoe does not fit everyone.

a reply to: rickymouse

In a medical system where intravenous vit C is regarded as howling at the moon I could not believe gene editing was being done in the US.

These scientists are taking a step, I hope it is forward.


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a reply to: rickymouse

I agree there is risk, but for so many people there is no choice or current hope.

If the people are willing, I don't see the harm? If it doesn't work, the willing patient may be harmed/killed, but it was there one chance to be normal/healthy and their choice. If it does work, they may be cured, and humanity a step closer to help thousands.

Just my 2 cents.

~Winter

a reply to: seasonal

I think this could potentially be groundbreaking. Hope everything goes well for the patient; I don't think I'd have the courage to try it. Flowers for Algernon comes to mind...

a reply to: sine.nomine

If the option was death, I could justify it and grow the courage.

I would imagine the patient feels like crap, is in pain, suffering and must know the next step is death. So what is there to loose?

originally posted by: seasonal
a reply to: sine.nomine

If the option was death, I could justify it and grow the courage.

I would imagine the patient feels like crap, is in pain, suffering and must know the next step is death. So what is there to loose?

Yeah, that's true. I'd probably roll those dice after mulling it over enough. It could help a lot of people. Hell, if it goes south, at least you're not stuck with the bill.

a reply to: sine.nomine

Good point, some one is paying for billions of copies of DNA. And by the time these cats are talking to you I would imagine you are in a bad way.

I bet the guy from the story is ready for it to work or for it to kill him. Or maybe a spider-man scenario.

originally posted by: Winterpain
a reply to: rickymouse

I agree there is risk, but for so many people there is no choice or current hope.

If the people are willing, I don't see the harm? If it doesn't work, the willing patient may be harmed/killed, but it was there one chance to be normal/healthy and their choice. If it does work, they may be cured, and humanity a step closer to help thousands.

Just my 2 cents.

~Winter

Most gene expression can be altered by diet or changing environmental factors. Some of the genetic traits cannot be effected by altering things, but most can. Medicines they use often work by altering of enzymes or proteins just as can be done by altering the diet. If you eat the wrong foods for your genetics, than the doctor makes money and the pharma companies get more money.

Can they edit my genes so they don't shrink in the dryer the first time they are washed?

That would really be something...

a reply to: seasonal

The potential gains far outweigh any risks involved.

Fact of the matter is that this type of technology taken to its logical conclusion could pretty much free humanity from disease, and possibly extend our lifespans by 100s of years.

If we can do a thing it generally follows that we do, do that thing.

It's kind of what we do, humanity simply has not, and probably never will, conceive a tool that we do not use.

originally posted by: madmac5150
Can they edit my genes so they don't shrink in the dryer the first time they are washed?

That would really be something...

Maybe it would be safer for these scientists to work on that than monkeying with our most critical system in our body.

To edit genes, you would have to edit all the genes in the body. How are they going to accomplish that, stem cells travel throughout the body regularly.

originally posted by: rickymouse

originally posted by: madmac5150
Can they edit my genes so they don't shrink in the dryer the first time they are washed?

That would really be something...

Maybe it would be safer for these scientists to work on that than monkeying with our most critical system in our body.

To edit genes, you would have to edit all the genes in the body. How are they going to accomplish that, stem cells travel throughout the body regularly.

No kidding... I am one edit away from being a politician.

The horror... the horror...

a reply to: rickymouse

That won't stop science progressing in the field.

Even if it's in some black budget facility off the books, if it can be done, then it probably is being done.

originally posted by: andy06shake
a reply to: rickymouse

That won't stop science progressing in the field.

Even if it's in some black budget facility off the books, if it can be done, then it probably is being done.

Which clearly explains the popularity of "Judge Judy"...

a reply to: madmac5150

Well, nothing is really clear these days, it's all generally opaque or obscured by design really.

Especially so in the field of ethically questionable technologies.

Look at it this way, the popularity of "Judge Judy", means that people are enthralled in her bullcrap rather than paying attention to whats happening under there very nose.

You look one way, "They" go the other.

a reply to: andy06shake


First and foremost this tech will be used to kill people. New tech is driven by 2 things profit and killing. Sad but true.

a reply to: seasonal

Yes we do indeed have a tendency to weaponize technology, or even create it for that very said purpose.

Again that problem lies with humanities penchant for destruction and until there is a paradigm shift that takes us in the opposite direction said problem is going to stay with us.

On the flip side, it could be argued that said penchant for destruction is what keeps us at the top of the food chain, all through its not it not my own personal feeling on the matter.

a reply to: andy06shake

I wish we were wired differently, but we are for real hell bent to destroy the enemy. And we always need an enemy.

a reply to: seasonal

Being an Apex predator has its downfalls.

We are indeed our own worst enemy, to date, but the universe is a big place, given enough time, im sure humanity will encounter a something worse than itself, that's if we managed to stay around and/or ever get the hell off this rock.