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#1
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A Targeted Genetic Cure For Muscular Dystrophy
Someone's going to make billions off this drug. It seems like the problem was the simplest in its class though. |
#2
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Apparently this protein cure is much more general than this. It seems to target "nonsense mutations", which is when the creation of a specific protein is hampered by an inadvertant "stop message" that shouldn't be there. This therapy might apply to a very wide range of diseases.
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#3
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It does look like a nice breakthrough, but scientists can cure almost anything in mice. It will be interesting to see how well this treatment works once the human studies start.
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