Invention Grant
- Patent Title: Splice-region antisense composition and method
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Application No.: US15723966Application Date: 2017-10-03
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Publication No.: US10533174B2Publication Date: 2020-01-14
- Inventor: Patrick L. Iversen , Robert Hudziak
- Applicant: Sarepta Therapeutics, Inc.
- Applicant Address: US MA Cambridge
- Assignee: SAREPTA THERAPEUTICS, INC.
- Current Assignee: SAREPTA THERAPEUTICS, INC.
- Current Assignee Address: US MA Cambridge
- Agency: Lathrop Gage LLP
- Agent Brian C. Trinque; Alan W. Steele
- Main IPC: C07H21/02
- IPC: C07H21/02 ; C07H21/04 ; C12N15/113

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.
Public/Granted literature
- US20180237773A1 SPLICE-REGION ANTISENSE COMPOSITION AND METHOD Public/Granted day:2018-08-23
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