For those who are interested, here is an interesting article on iPSC stem cells used for research and drug development of genetic diseases, which I think could be the new and ideal way of trialing antisense compounds.
http://www.sciencedirect.com/science/article/pii/S0092867408010015
The MS stem cells on a dish I mentioned earlier:
http://www.smh.com.au/technology/sci-tech/scientists-unveil-first-ms-stem-cell-model-20111221-1p5l4.html.
This will save cost and time with almost unliited trials without human safety issues.
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