Details for Patent: 9,416,361

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Summary for Patent: 9,416,361

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):	Iversen; Patrick L. (Corvallis, OR), Hudziak; Robert (Blodgett, OR)
Assignee:	Sarepta Therapeutics, Inc. (Cambridge, MA)
Application Number:	14/535,098
Patent Claim Types: see list of patent claims	Compound;

Drugs Protected by US Patent 9,416,361

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