1. Great News! - for only some of the boys
    Bob Getler Bob Getler United States says:
    January 10, 2011 at 12:40 PM

    I believe there are 79 exons in scope for DMD.  Fantastically there are some Exons actively being targeted for skipping (eg, 50, 51, 44).

    While this is great news for those who can benefit from skipping these relatively few Exons, I suspect the majority of boys can *not* benefit until many additional Exons can also be skipped.   Some boys even need multiple Exons to be skipped.    Some need such huge areas to be skipped the resulting Dystrophin protein may not be of great value.

    It is great news but I think we need to be mindful to mention the percentage of boys who may benefit, the percentage who will not, and not generate an impression that this great technology is of universal use to all the suffering, young boys.

    Thank you.

    Reply
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