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 2519863
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 avec lequel un exon peut être sauté dans un ARNm prémessager et donc exclu d'un ARNm produit. L'invention concerne également des méthodes pour modifier la structure secondaire d'un ARNm et interférer dans des processus d'épissage et des utilisations des oligonucléotides, et de méthodes destinées au traitement de maladies. L'invention concerne en outre des compositions pharmaceutiques, des méthodes et des moyens pour induire le saut de plusieurs exons dans un ARNm prémessager.
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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