Details for Patent: 10,533,174
Summary for Patent: 10,533,174
| Title: | Splice-region antisense composition and method |
| 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. |
| Inventor(s): | Patrick L. Iversen, Robert Hudziak |
| Assignee: | Sarepta Therapeutics Inc |
| Application Number: | US15/723,966 |
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Patent Claim Types: see list of patent claims | Composition; Compound; |
Drugs Protected by US Patent 10,533,174
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International Family Members for US Patent 10,533,174
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