Invention Grant
US08759507B2 Modulation of exon recognition in pre-mRNA by interfering RNA structure 有权
通过干扰RNA结构调节前mRNA的外显子识别

Modulation of exon recognition in pre-mRNA by interfering RNA structure
Abstract:
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 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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