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MODULATION OF EXON RECOGNITION IN PRE-MRNA BY INTERFERING WITH THE BINDING OF SR PROTEINS AND BY INTERFERING WITH SECONDARY RNA STRUCTURE.
MODULATION OF EXON RECOGNITION IN PRE-MRNA BY INTERFERING WITH THE BINDING OF SR PROTEINS AND BY INTERFERING WITH SECONDARY RNA STRUCTURE.
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机译:通过干扰SR蛋白的结合和干扰二级RNA结构来调节前MRNA中外显子的识别。
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摘要
The invention provides a method for generating an oligonucleotide with which an exon may be skipped in a pre-mRNA and thus excluded from a produced mRNA thereof. Further provided are methods for altering the binding of an SR protein and/or methods for altering the secondary structure of an mRNA to interfere with splicing processes and uses of the oligonucleotides and methods in the treatment of disease. Further provided are pharmaceutical compositions and methods and means for inducing skipping of several exons in a pre-mRNA.
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