C - Chemistry – Metallurgy – 07 – H
Patent
C - Chemistry, Metallurgy
07
H
C07H 21/00 (2006.01) A61K 31/70 (2006.01) A61K 31/7088 (2006.01) A61K 48/00 (2006.01) C07H 21/02 (2006.01) C12N 15/11 (2006.01) C12N 15/67 (2006.01) C07K 14/805 (2006.01)
Patent
CA 2162361
A method of combatting aberrant splicing in a pre-mRNA molecule containing a mutation is disclosed. When present in the pre-mRNA, the mutation causes the pre-mRNA to splice incorrectly and produce an aberrant mRNA or mRNA fragment different from the mRNA ordinarily encoded by the pre-mRNA. The method comprises hybridizing an antisense oligonucleotide to the pre-mRNA molecule to create a duplex molecule under conditions which permit splicing. The antisense oligonucleotide is one which does not activate RNase H, and is selected to block a member of the aberrant set of splice elements created by the mutation so that the native intron is removed by splicing and the first mRNA molecule encoding a native protein is produced. Oligonucleotides useful for carrying out the method are also disclosed.
Dominski Zbigniew
Kole Ryszard
Sim & Mcburney
University Of North Carolina At Chapel Hill
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