Giving mice Down’s syndrome
A “technical” step forward has been made in understanding Down’s syndrome: researchers have successfully introduced 90% of the 250 genes on human chromosome 21 into the embryonic stem cells of mice. A normal person has two copies of chromosome 21, but a person with Down’s syndrome has three, making it part of the aneuploidy class of disorders (of which I have touched on in the past). Using the compromised stem cells, researchers were able to create a strain of mice that carried the extra chromosome.
This strain of mice has problems with memory, brain function, and heart formation. It remains to be seen whether they show a disposition towards other diseases like leukemia, which people with Down’s often have to deal with. By creating the modified stem cells, researchers hope to learn which genes control which aspects of the Down’s symptom. For instance, by learning which genes affect heart formation, they hope to one day create stem cell therapies that will turn off the genes of developing children with Down’s.
While far from a cure, this ability to create strains of mice that can be experimented with shows huge promise in the field of Down’s research. If scientists can discover which genes control brain and heart formation, and which genes alter thyroid function, they can potentially suppress their expression in developing fetuses at some point down the road.
This, of course, is assuming that such therapies will be legal in places like the United States, whose laws are quite restrictive when it comes to stem cell research. I suppose that one could go to a place like the UK for treatment, but I suspect that if real-word promise is offered for relatively mainstream conditions, conservative public opinion in the US will change. Slowly.
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