Granted Patent
B2
US 8,759,507 · App. 12/976,381 · Granted Jun 24, 2014
Modulation of exon recognition in pre-mRNA by interfering RNA structure
Assignee:
Academisch Ziekenhuis Leiden
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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.
Claims (2)
1. An oligonucleotide consisting of SEQ ID NO. 27, wherein each ribose moiety of said oligonucleotide is 2′—O—methyl substituted.
2. The oligonucleotide of claim 1 , wherein each internucleoside linkage of said oligonucleotide is a phosphorothioate internucleoside linkage.
Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST
Recorded Jan 31, 2013
From: VAN DEUTEKOM, JUDITH CHRISTINA THEODORA, DR.
To: ACADEMISCH ZIEKENHUIS LEIDEN
Reel/Frame 029733/0786 →
Continuity (4)
Continuation
PCTNL0300214
· Mar 21, 2003