Method to Turn Off Down Syndrome DNA?

[LongBlade]

A promising study has confirmed in theory that it is possible to turn off the extra chromosome that causes Down Syndrome.

In a new study published online in the journal Nature, scientists from University of Massachusetts Medical School (UMMS) harnessed the abilities of a naturally occurring gene called XIST that acts as an "off switch" in X chromosomes.  In a culture of stem cells, the researchers were able to repurpose the XIST gene so that instead of silencing X chromosomes, it silenced the extra chromosome 21 instead.

Though the research only shows proof-of-principle for turning off the chromosome – meaning the method is a long way from being utilized in humans – the findings have huge implications for the future of Down syndrome research. Researchers hope this study will pave the way for a better understanding of the disorder's pathology and potentially help to create new therapeutic targets for therapies.

Read more: http://www.foxnews.com/health/2013/07/17/scientists-find-way-to-silence-extra-chromosome-that-causes-down-syndrome/?intcmp=obnetwork#ixzz2ZKd2ypsh

[eyebiter]

What happens if an adult with Down Syndrome receives this treatment?  Would it be a permanent fix or a 'Flowers for Algernon" scenario where they improve, plateau, then regress over time?

[LongBlade]

What happens if an adult with Down Syndrome receives this treatment?  Would it be a permanent fix or a 'Flowers for Algernon" scenario where they improve, plateau, then regress over time?

I don't think so. At this point I think the change is only theoretical, and then as the child is forming.

[Huw the Poo]

Yeah, the therapy would have to begin pretty damn early in the embryonic development stage for this to be much use, I would think.  Still...great news, of that there can be no doubt.

Edit: Hmm, I've just read the article and it doesn't say how they would target the extra chromosome in vivo.  That's gonna take some doing.  Also, I don't think the Fox reporter knows much about biology:

The XIST gene makes a unique non-coding RNA, which accumulates in the nucleus of the cell of the chromosome

Lolwut

[Staggerwing]

Maybe he thinks there is a terrorist cell hiding in the chromosome. You can never be too careful, am I right?

Or maybe he feels that the word order is not important. After all, what could happen if you accidentally switched a few words around? Funny words like guanine, adenine, thymine, and cytosine are all pretty interchangeable. No need to get picky is there?

[Huw the Poo]

After all, what could happen if you accidentally switched a few words around? Funny words like guanine, adenine, thymine, and cytosine are all pretty interchangeable. No need to get picky is there?

Haha!  Yeah, they're all practically the same; nobody will notice. :D

[undercovergeek]

The last time I read about a cure for DS there was a lot of people up in arms and rather a lot of DS 'sufferers' saying that one was trying to wipe them out - I didn't quite understand it, particularly with a large majority of the parents saying if they had to do it all again they'd still have their DS baby - unbelievable