C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 15/11 (2006.01) A61K 31/7088 (2006.01) A61K 48/00 (2006.01)
Patent
CA 2524255
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.
L'invention concerne un procédé de génération d'un oligonucléotide à l'aide duquel un exon peut être sauté dans un pré-ARNm et ainsi exclu de son ARNm produit. L'invention concerne également des méthodes de modification de la structure secondaire d'un ARNm pour interférer avec les processus d'épissage ainsi que les utilisations des oligonucléotides et des méthodes dans le traitement de maladies. L'invention concerne en outre des compositions pharmaceutiques ainsi que des méthodes et des moyens induisant le saut de plusieurs exons dans un pré-ARNm.
Aartsma-Rus Annemieke
Den Dunnen Johannes Theodorus
Van Deutekom Judith Christina Theodora
Van Ommen Garrit-Jan Boudewijn
Academisch Ziekenhuis Leiden
Borden Ladner Gervais Llp
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