Invention Grant
- Patent Title: Method for efficient exon (44) skipping in duchenne muscular dystrophy and associated means
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Application No.: US14859598Application Date: 2015-09-21
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Publication No.: US10246707B2Publication Date: 2019-04-02
- Inventor: Gerard Johannes Platenburg , Josephus Johannes de Kimpe , Judith Christina Theodora van Deutekom , Garrit-Jan Boudewijn van Ommen , Annemieke Aartsma-Rus
- Applicant: BioMarin Technologies B.V. , Academisch Ziekenhuis Leiden
- Applicant Address: NL Leiden NL Leiden
- Assignee: Biomarin Technologies B.V.,Academisch Ziekenhuis Leiden
- Current Assignee: Biomarin Technologies B.V.,Academisch Ziekenhuis Leiden
- Current Assignee Address: NL Leiden NL Leiden
- Agency: Jones Day
- Priority: EP08156193 20080514
- Main IPC: C12N15/11
- IPC: C12N15/11 ; C12N15/113 ; A61K48/00
Abstract:
The invention relates to a nucleic acid molecule that binds and/or is complementary to the nucleotide molecule having sequence 5′-GUGGCUAACAGAAGCU (SEQ ID NO 1) and to its use in a method for inducing skipping of exon 44 of the DMD gene in a DMD patient.
Public/Granted literature
- US20160053262A1 METHOD FOR EFFICIENT EXON (44) SKIPPING IN DUCHENNE MUSCULAR DYSTROPHY AND ASSOCIATED MEANS Public/Granted day:2016-02-25
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