SPLICE-REGION ANTISENSE COMPOSITION AND METHOD

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United States of America Patent

SERIAL NO

15205722

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Abstract

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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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Patent Owner(s)

Patent OwnerAddress
SAREPTA THERAPEUTICS INC215 FIRST STREET CAMBRIDGE MA 02142

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Inventor(s)

Inventor Name Address # of filed Patents Total Citations
Hudziak, Robert Blodgett, US 8 613
Iversen, Patrick L Corvallis, US 139 5121

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