Scientists Reverse Muscular Dystrophy In Dogs

Al writes "Scientists have taken a step toward developing a cure for Duchenne muscular dystrophy (DMD) by successfully treating the condition in dogs using a novel genetic technique. The scientists used a method called exon skipping, which involves adding a genetic 'patch' to block transcription of a portion of the gene involved in DMD. This puts the remaining genetic sequence back in order, essentially creating a much less severe version of the condition. The scientists recorded some remarkable video footage showing the resulting improvements in several dogs with naturally-occurring DMD. More work is needed before the treatment can be given to humans, however, because DMD sufferers often have different genetic mutations."

Beware

[dnormant]

The video link is pop up hell in IE.

Re:Is it heritable?

[Anonymous Coward]

I'd be interested to see whether or not the "patch" is heritable; the article doesn't mention it. In any case, it's really impressive work.

It's not.

Great News

[Nos.]

I just found out that two nephews of three are positive for DMD. This basically confirms that my sister-in-law is a carrier. We're in the middle of trying to determine if my wife is a carrier, and thus if our two sons are at risk. To say the least this is a very stressful time in our lives, and there are no quick answers. However, seeing a big jump like this in treatment is great news.

Re:Is it heritable?

[PotatoFarmer]

I guess it would depend on the potential methods of inheritance. If, in addition to directly modifying the production of male sperm, the patch could be delivered through the placenta to a fetus similar to how antibodies are transferred then it could still be heritable through the female.

That all being said, I'm not a biologist, so it's entirely possible that what I've described can't actually happen.

Re:Is it heritable?

[vivin]

IANAG (IANA Geneticist), but from what little I know about genetics, I doubt it is heritable. The only way something can be heritable is if it modifies any of the germ cells (sperm or ova). In fact, some of the "junk" DNA that we have are actually inactive sequences of ancient retroviruses (ERVs - Endogenous retroviruses [wikipedia.org]) that infected the germ cells in our ancestors.

Re:Great News

[anderesa]

Dear Andrew,
as a daily reader of Slashdot and also father of four young kids, two of them having Duchenne, I'm surprised that this terrible desease is discussed among this community.

Unfortunately, the discussion doesn't go very deep with few interesting threads. I cross my fingers for your family. My wife also is a carrier but my two sister-in-laws. In fact, we found out that my wife got the defect (3 Exons are deleted on one X-chromosom) from her mother but she's been the only one among five kids to inherit the defect X-chromosom. I've also read in several places that the odds of being a carrier are 66% if one of your kids has the genetic defect

Last, I'd like to point you to some great reports about latest research incl. exon skipping techniques. They are all written in easy language understandable by parents and other people without a PhD in biochemistry.
http://www.duchenne-information.eu/home-en.htm

Take care
--Sam