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, Adriana Marie Rus, Gerard Johannes Platenburg, Judith Christina Theodora Van Deutekom, 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 the exon. The invention further relates to such molecule used in the method.
Claims23
An antisense oligonucleotide whose base sequence consists of the base sequence of 5 ′UUUGCCGCUGCCCAAUGCCAUCCUG-3′ (SEQ ID:
- NO:
- 3), said oligonucleotide comprising a modification.
Dependencies are inferred from the claim text.