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US 2012/0059042 A1Published application
Method for efficient exon (44) skipping in duchenne muscular dystrophy and associated means
AssigneesProsensa 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.
Claims11
A nucleic acid molecule that binds and/or is complementary to a nucleotide having nucleotide sequence:
- (SEQ ID NO 1)
- 5′-GUGGCUAACAGAAGCU.
Dependencies are inferred from the claim text.