METHODS AND MEANS FOR EFFICIENT SKIPPING OF EXON 45 IN DUCHENNE MUSCULAR DYSTROPHY PRE-mRNA
First Claim
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1. An isolated antisense oligonucleotide consisting of 22, 23, 24, 25, 26, 27, 28 or 29 nucleotides, wherein said oligonucleotide is complementary along its entire length to a sequence in part of the human dystrophin exon 45 pre-mRNA, wherein said sequence is complementary to at least 22 nucleotides of a sequence consisting of 5′
- -UUUGCCGCUGCCCAAUGCCAUCCUG-3′
(SEQ ID NO;
3).
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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 isolate 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.
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8 Claims
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1. An isolated antisense oligonucleotide consisting of 22, 23, 24, 25, 26, 27, 28 or 29 nucleotides, wherein said oligonucleotide is complementary along its entire length to a sequence in part of the human dystrophin exon 45 pre-mRNA, wherein said sequence is complementary to at least 22 nucleotides of a sequence consisting of 5′
- -UUUGCCGCUGCCCAAUGCCAUCCUG-3′
(SEQ ID NO;
3). - View Dependent Claims (2, 3, 4, 5, 6, 7, 8)
- -UUUGCCGCUGCCCAAUGCCAUCCUG-3′
Specification