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US 10,246,707 B2Granted
Method for efficient exon (44) skipping in duchenne muscular dystrophy and associated means
AssigneesBioMarin Technologies B.V., Academisch Ziekenhuis Leiden
InventorsGerard Johannes Platenburg, Josephus Johannes de Kimpe, Judith Christina Theodora van Deutekom, Garrit-Jan Boudewijn van Ommen, Annemieke Aartsma-Rus
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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.
Claims12
A single stranded antisense oligonucleotide 16 to 25 nucleotides in length that comprises a base sequence of the sequence selected from the group consisting of SEQ ID NO:
- 5 to SEQ ID NO:
- 16, SEQ ID NO:
- 20 to SEQ ID NO:
- 28, SEQ ID NO:
- 30 to SEQ ID NO:
- 34, SEQ ID NO:
- 41, and SEQ ID NO:
- 46, wherein the antisense oligonucleotide induces skipping of exon 44 of human dystrophin pre-mRNA, and comprises a modification.
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Dependencies are inferred from the claim text.