First Successful Use of Genome Editing In Living Animals Cures Hemophilia In Mice

[n2doc]

By Rebecca BoylePosted 06.27.2011 at 12:02 pm

A targeted snip through DNA’s double helix can take out a mutated gene that causes hemophilia, curing mice of the disease, a new study found. It’s the first study to use this form of genome editing in a living animal, and it could have implications for genetic treatment of other diseases, notably AIDS.

Scientists say the research is a major step forward for gene therapy, which has long promised to cure disease by editing genetic sequences.

The therapy is based on enzymes called zinc-finger nucleases, which serve as a sort of genetic scissors. The enzymes are engineered to match a specific gene location on a chromosome, where they snip through DNA’s double helix.

...

The study shows that zinc finger proteins and replacement genes can be used to induce changes in living animals, which is promising for a wide range of therapies. For instance, other researchers are using ZFNs to disrupt a gene that makes a receptor used by the AIDS virus, as the New York Times reports. People without that gene, CCR5, are naturally immune to HIV.

article

http://www.popsci.com/science/article/2011-06/genome-repair-kit-cures-blood-disorder-mice

[provis99]

1. cool.

Maybe the next step is trying to cure congenital stupidity in conservatives.

[murray hill farm]

2. Seriously impressive!

Gene therapy will be the answer to so many illnesses...and new gene therapies are coming along quickly now.

[Donnachaidh]

3. no offense -- but cure HEMOPHILIA in people first fer crissakes!

If it's working in mice take it the next damned step and cure these kids!

[laconicsax]

5. Umm...this was a test to see if it works.

Better to test an unproven method of genetic manipulation in mice first BEFORE using it in children.

[Donnachaidh]

6. and it worked, yes?

Soooo, the next step would be to cure hemophiliacs, yes? As the parent of a child with that affliction I've been told since before he was born this would be coming.

But this piece is talking about using it for other problems -- potentially leapfrogging those kids who have been waiting. Most likely because there are more glamorous afflictions this can be applied to.

Those kids need to be helped. Period.

[laconicsax]

7. No, the next step is to conduct safety and efficacy trials in humans.

If you skip steps, there could be potentially horrible results. This method can't be approved for use in treating humans before it's shown to be both effective AND safe. We're talking about changing someone's DNA here; the consequences of rushed work could be horrific.

Hemophilia isn't the only genetic disorder that affects children. Having an effective delivery method could mean cures for more than just hemophilia and the author of the article would be negligent if she didn't address the wide variety of conditions that could be benefited by this.

[laconicsax]

4. Looks like a good time to start the conversation about the broader implications.