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US 8,759,507 B2Granted
Modulation of exon recognition in pre-mRNA by interfering RNA structure
AssigneeAcademisch Ziekenhuis Leiden
InventorJudith Christina Theodora Van Deutekom
Life of this patent
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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.
Claims2
An oligonucleotide consisting of SEQ ID NO. 27, wherein each ribose moiety of said oligonucleotide is 2′—O—methyl substituted.
Dependencies are inferred from the claim text.