Antisense oligonucleotides which combat aberrant splicing and me

Details Antisense oligonucleotides which combat aberrant splicing and me Antisense oligonucleotides which combat aberrant splicing and me

1995-05-30

1997-05-06

Rories, Charles C. P.

Organic compounds -- part of the class 532-570 series

Organic compounds

Carbohydrates or derivatives

536 243, 536 2431, 536 2433, 536 245, 435 6, 424450, C07H 2100, A61K 4800, C12Q 168

Patent

active

056272740

ABSTRACT:
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.

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