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Down syndrome is caused by genetic disorder when in DNA chromosome #21 has three copies (instead of 2 copies). If I understand correctly then the Genetic Engineering is the closest field in modern medicine that studies how to alter host's DNA.

Would there in next few years be a cure for Down's syndrome (i.e. remove the third copy of chromosome #21 in an adult's DNA)? If not, then what are difficulties that would still need to be overcome in genetic engineering to do something like that?

For example, is it impossible to consistently change DNA for all cells in body? Would these cells start to "attack" each other while being in partially reconfigured state?). If I understand correctly then CRISPR* method is the new tool in genetic engineering that may allow to do such DNA manipulations?

P.S. as it may be obvious from my question I don't have medical background and my questions is motivated by having relative affected by Down's syndrome.

P.P.S. I found this link http://www.prnewswire.com/news-releases/new-down-syndrome-therapy-discovered-300135102.html - not sure how to interpret it.

  • That's a very nice question! – user4845 Jul 11 '16 at 0:00
  • This is a good question but I think it's too broad. Just answering any one of the three questions you posed would make a good chunk of work. Recommend you edit it down to #1 and save the other two for subsequent questions. – Carey Gregory Jul 11 '16 at 3:25
  • @CareyGregory I reduce scope of the question. Thanks for the feedback! – john1234 Jul 15 '16 at 1:39
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Reversing Down Syndrome in adults is not something being currently researched to my knowledge. Alerting the DNA throughout the body of a person at this point is purely science fiction, but even if it were possible the critical structures that were created due to the genetic mutation wouldn't just go away.

Take the simian crease in the hand of many with down syndrome. If you altered the DNA of the entire person they'd still have the simian crease because it was established early in the early formation of the person as an embryo. The genes for creating a hand aren't re-expressed later.

More importantly, many of the brain structures and pathways are formed in early childhood and puberty and a genetic change after then would have little effect.

Genetic alteration research would first be focused on embryonic intervention, but in not aware of any studies actively looking at that even. Most research is focused on early detection, causes/prevention, and therapeutic intervention for mitigation of symptoms. You can see none of the studies currently being conducted by the National Down Syndrome Society or the International Mosaic Down Syndrome Society are related to genetic intervention. These may also give you a feel for the type of research (here, here, and here).

  • I found this link - prnewswire.com/news-releases/… - where apparently there is a company working on this. Though, haven't been able to Google up any updates from them so not sure how legit reason for hope that is. – john1234 Jul 15 '16 at 1:43
  • This is almost a good answer. If you added a citation or two to support your statements it would be a good answer. – Carey Gregory Jul 15 '16 at 15:03
  • @CareyGregory - does that satisfy your desire for references? – Jared Jul 16 '16 at 14:50
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    It's not my desire; it's the site's desire. But yes, much better. :-) – Carey Gregory Jul 16 '16 at 17:22
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    Since health is an important topic, the site has a strict policy that all answers should be backed up with reliable references in order to provide the community with the means to assess the merit of the answer, regardless of the reader's background. @CareyGregory can't really do anything else than request references as this is how the community decided. – Narusan Apr 15 '17 at 13:18

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