C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 15/11 (2006.01) A61K 31/7088 (2006.01)
Patent
CA 2605512
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.
L'invention concerne un procédé permettant de produire un oligonucléotide au moyen duquel un exon peut être sauté dans un pré-ARNm et exclu par conséquent d'un ARNm produit à partir de celui-ci. De plus l'invention concerne des procédés permettant de modifier la liaison d'une protéine SR et/ou des procédés permettant de modifier la structure secondaire d'un ARNm, aux fins d'interférence avec des procédés d'épissage et des utilisations des oligonucléotides et des procédés dans le traitement de maladie. L'invention concerne enfin des compositions pharmaceutiques et des procédés et des moyens permettant d'induire le saut de plusieurs exons dans un pré-ARNm.
Aartsma-Rus Annemieke
Van Deutekom Judith Christina Theodora
Van Ommen Garrit-Jan Boudewijn
Academisch Ziekenhuis Leiden
Borden Ladner Gervais Llp
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