Sceintists use HIV to cure a brain disease.

[Vendog]

New gene therapy halts 2 boys' rare brain disease

WASHINGTON – French scientists mixed gene therapy and bone marrow transplants in two boys to seemingly halt a brain disease that can kill by adolescence. The surprise ingredient: They disabled the HIV virus so it couldn't cause AIDS, and then used it to carry in the healthy new gene.

The experiment marks the first time researchers have tried that long-contemplated step in people — and the first effective gene therapy against a severe brain disease, said lead researcher Dr. Patrick Aubourg of the University Paris-Descartes.

Although it's a small, first-step study, it has "exciting implications" for other blood and immune disorders that had been feared beyond gene therapy's reach, said Dr. Kenneth Cornetta, president of the American Society of Gene and Cell Therapy.

"This study shows the power of combining gene therapy and cell therapy," added Cornetta, whose own lab at Indiana University has long researched how to safely develop gene delivery using lentiviruses, HIV's family.

The research was published in Friday's edition of the journal Science.

In 20 years of gene therapy research, there have been few home runs and some headline-making setbacks — including a risk of leukemia caused by otherwise successful gene therapy for another rare disorder, "bubble boy disease." That's a risk that specialists hope a lentivirus-based gene therapy will eliminate.

Best known from the movie "Lorenzo's Oil", adrenoleukodystrophy, or ALD, is a rare genetic disease that, in its most devastating form, destroys the coating of nerve fibers in boys' brains. Without that coating, called myelin, the neurological system breaks down. The disease typically strikes between the ages of four and 10, leading to blindness, deafness, dementia and loss of muscle control, and killing them within a few years.

Bone marrow transplants can halt ALD by letting new myelin-forming stem cells take root. But it's difficult to find a matching marrow donor, and the transplant itself is very risky.

So what if stem cells from the boys' own bone marrow could be genetically corrected, eliminating the ALD mutation? To do that, Aubourg's team had to overcome a technical hurdle: Gene therapy works when scientists harness deliver a healthy new gene by attaching to a virus that can harmlessly infect cells. But none of today's so-called gene therapy "vectors" could penetrate enough of the stem cells needed for an ALD treatment to work.

Unlike most viruses, HIV can penetrate stem cells, and it sticks permanently. So Aubourg's team removed the genetic parts of HIV that make it dangerous, leaving basically a scaffolding to carry the new therapeutic gene.

Then they culled stem cells from two 7-year-old boys in the early stages of ALD, and mixed in the healthy gene. The boys underwent bone marrow-destroying chemotherapy and then had their genetically corrected stem cells reinserted.

Two years later, the boys have shown no sign of worsening brain damage and are functioning well with 15 percent of their blood cells producing the healthy protein, said Aubourg, who plans to test the experimental procedure in more patients. An advocacy group, the Stop ALD Foundation, is working to raise money for a similar U.S. study.

[Jeryhn]

Sounds pretty cool, but I kinda wonder how they neutered the HIV virus more and what other practical applications it can have.

[Restrat]

viruses have a small single stranded genome that is very easy to cut up and rearrange however you wish.
You can do it in an introductory biology class in college in two days

[Ohemgee]

That lab was so long

[Chun Liroy]

And this is why I want to become a virologist

[Yuri-G]

everyone start lining up for blood transfusions, I'll be donating all day for a nominal (read: very large) fee!

in all seriousiness, this is pretty cool.

[Chun Liroy]

Blood transfusions? Why don't you at least do it the fun way~!

[Xno Kappa]

Hmmm. Seems like gay people can finally contribute to a society of values.

[Tristam]

Sounds pretty cool, but I kinda wonder how they neutered the HIV virus more and what other practical applications it can have.

They get rid of the DNA that allows the virus to replicate, but leave the part of the genome that allows the virus to integrate part of its DNA into the host DNA.

[Vuitton]

Hmmm. Seems like gay people can finally contribute to a society of values.

Taint licking is a value society should have already held. America is so backwards.

[Maguspk]

So basically they used HIV as a sort of Glad™ container for the stem cells, doused the kids with chemotherapy, then safely reinserted the stem cells? That's really fucking pro.

[Vuitton]

So basically they used HIV as a sort of Glad™ container for the stem cells, doused the kids with chemotherapy, then safely reinserted the stem cells? That's really fucking pro.

They also had to burp the viruses to ensure the stem cells stayed fresh.

[eunhye]

No one's mentioned the T-Virus? jeesh. Sounds a lot like it if you asked me, super creepy.

[Day]

Taint licking is a value society should have already held. America is so backwards.

Taint licking isn't exclusive to homosexuals, just thought I'd point that out.

[Yuri-G]

Blood transfusions? Why don't you at least do it the fun way~!

hey now, I never said that wasn't an option!

[Xno Kappa]

What the fuck is taint licking?

[Day]

Getting your taint licked and/or licking taint. It's pretty self explanatory. Feels good man.

[Xno Kappa]

Licking butt-holes?

[Day]

Close. But taint is area above ye anus. Rimjobs are another story completely, but that's for another thread I believe.

[Silenka]

Gene therapy is an amazing thing, once the kinks are worked out of the system (gene delivery and minimization of risk) we'll be curing everything from HIV to cancer (or at least putting it in severe remission). I can't wait until this gets more widespread/mainstream.