Splice-region antisense composition and method
US8436163B2 · kind B2 · utility
38Cited by
43References
7Claims
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Key dates
| Filing date | Aug 26, 2011 |
| Grant date | May 7, 2013 |
| Priority date | — |
| Expiry date | Aug 26, 2031 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2320/33
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
Antisense compositions targeted against an mRNA sequence coding for a selected protein, at a region having its 5′ end from 1 to about 25 base pairs downstream of a normal splice acceptor junction in the preprocessed mRNA, are disclosed. The antisense compound is RNase-inactive, and is preferably a phosphorodiamidate-linked morpholino oligonucleotide. Such targeting is effective to inhibit natural mRNA splice processing, produce splice variant mRNAs, and inhibit normal expression of the protein.
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