Splice-region antisense composition and method
US8895722B2 · kind B2 · utility
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10Claims
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Key dates
| Filing date | Apr 9, 2013 |
| Grant date | Nov 25, 2014 |
| Priority date | — |
| Expiry date | Apr 9, 2033 |
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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