Antisense oligonucleotides which combat aberrant splicing and methods of using the same
US5627274A · kind A · utility
Assignee
Inventors
Key dates
| Filing date | May 30, 1995 |
| Grant date | May 6, 1997 |
| Priority date | — |
| Expiry date | May 30, 2015 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2310/3521
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
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.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.