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The introduction of the small RNAs will produce a protein that is somewhat shortened because an exon is skipped and some amino acids are missing, but it may still result in a protein that has some function. The small RNAs, antisense RNAs, used for exon skipping are complementary to bases in the premRNA, which will prevent proper associating of spliceosome for intron removal. A Goyenvalle et al Science :
In order to skip the mutated exon and potentially treat DMD select the best antisense RNA targets.
a splice site of the intron upstream of the exon to be skipped
a branch point of the intron upstream of the exon to be skipped
a splice site of the intron upstream of the exon to be skipped
a splice site of the intron downstream of the exon to be skipped
Question Source: Pierce eGenetics: A Conceptual Approach Publisher. WH Freemn
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