Splice-region antisense composition and method
US8067569B2 · kind B2 · utility
51Cited by
37References
20Claims
0Family size
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Key dates
| Filing date | May 11, 2006 |
| Grant date | Nov 29, 2011 |
| Priority date | — |
| Expiry date | May 19, 2028 |
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 a phosphorodiamidate-linked morpholino oligonucleotide containing uncharged phosphorodiamidate linkages interspersed with cationic phosphorodiamidate linkages. Such targeting is effective to inhibit natural mRNA splice processing, produce splice variant mRNAs, and inhibit normal expression of the protein.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.