Which exon needs to be skipped and who is working it ?

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44 needs skipped. Both Prosensa and AVI will work on it. It is Prosensa's 2nd exon skipping drug, they plan trials at the moment.

http://www.parentprojectmd.org/site/DocServer/InterviewsKate_Judith...

On page 5:

"The skipping of which exon will you develop next, 44?
Yes, 44, we are about to start a systemic phase I-IIA
trial similar to the present 51-trial. We will not do a local,
intramuscular trial."
Will exon skipping also help the duplication of 45?
skipping 46 will also help those with deletion of 45. don't know when or who will do it first.
it is predicted that you would have to skip exon 45 to help a duplicate of 45. not sure how that would help. seems you would need to skip 44 and 45 or 45 and 46 to restore the reading frame. from what i understand, they are not yet working on skipping multiple exons...but i have been out of the loop for quite a few months now.

Amanda Rudd said:
Will exon skipping also help the duplication of 45?

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