Methods and means for efficient skipping of exon 45 in Duchenne muscular dystrophy pre-mRNA
AssigneesBioMarin Technologies B.V., Academisch Ziekenhuis Leiden
InventorsJosephus Johannes De Kimpe, Gerardus Johannes Platenburg, Judith Christina Theodora Van Deutekom, Annemieke Aartsma-Rus, Garrit-Jan Boudewijn Van Ommen
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Abstract
The invention relates to a method for inducing or promoting skipping of exon 45 of DMD pre-mRNA in a Duchenne Muscular Dystrophy patient, preferably in an isolated (muscle) cell, the method comprising providing an isolated muscle cell with a molecule that binds to a continuous stretch of at least 21 nucleotides within said exon. The invention further relates to such molecule used in the method.
Claims11
A pharmaceutical composition comprising an antisense oligonucleotide and an excipient selected from the group consisting of polyethylenimine, and polypropylenimine, wherein said antisense oligonucleotide consists of the base sequence:
- 5′-UUUGCCGCUGCCCAAUGCCAUCCUG-3′ (SEQ ID:
- NO:
- 3), said oligonucleotide comprising a modification.
Dependencies are inferred from the claim text.