Details for Patent: 8,895,722

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. (Corvallis, OR)
Filing Date:	Apr 09, 2013
Application Number:	13/859,518
Claims:	1. An antisense compound composed of a 2'-O-methyl modified backbone and a base sequence of 12 to 25 nucleotide bases which is complementary to a target region within one exon of multiple exons of a preprocessed mRNA encoding a human protein, wherein the 5'-end of the target region is 12 bases downstream of a normal splice acceptor site in said preprocessed mRNA. 2. The compound of claim 1, which is about 24 nucleotide bases. 3. A composition, comprising an antisense compound of claim 1 and a pharmaceutical carrier. 4. The composition of claim 3, formulated for intramuscular delivery. 5. The composition of claim 3, formulated for intravenous (IV) infusion. 6. The composition of claim 3, formulated for subcutaneous delivery. 7. A method of producing a splice variant mRNA in a human subject, comprising administering to the subject an antisense compound of claim 1, wherein the antisense compound alters splicing of a preprocessed mRNA encoding a human protein, to produce a variant mRNA, wherein the variant mRNA expresses a variant protein. 8. The method of claim 7, wherein said administering is by intramuscular delivery. 9. The method of claim 7, wherein said administering is by intravenous (IV) infusion. 10. The method of claim 7, wherein said administering is by subcutaneous delivery.