Splice-region antisense composition and method
First Claim
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1. An antisense compound having an uncharged morpholino backbone and the base sequence presented as SEQ ID NO:
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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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6 Claims
- 1. An antisense compound having an uncharged morpholino backbone and the base sequence presented as SEQ ID NO:
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