Exon skipping for Exon 45 deletion
Which exon needs to be skipped and who is working it ?
Replies to This Discussion
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Permalink Reply by Ofelia Marin on
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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."
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Permalink Reply by Amanda Rudd on
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Will exon skipping also help the duplication of 45?
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Permalink Reply by MicahsDaddy on
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skipping 46 will also help those with deletion of 45. don't know when or who will do it first.
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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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