SPLICE-REGION ANTISENSE COMPOSITION AND METHOD
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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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Citations
41 Claims
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1-38. -38. (canceled)
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39. 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 an exon of multiple exons of a preprocessed mRNA encoding a human protein, wherein the 5′
-end of the target region is 6 or 7 bases downstream of a normal splice acceptor site in said preprocessed mRNA. - View Dependent Claims (40, 41)
- -O-methyl modified backbone and a base sequence of 12 to 25 nucleotide bases which is complementary to a target region within an exon of multiple exons of a preprocessed mRNA encoding a human protein, wherein the 5′
Specification