Antisense nucleic acids
First Claim
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1. An antisense oligomer which causes skipping of the 55th exon in a human dystrophin gene, wherein the base sequence of the antisense oligomer consists of the base sequence of (i) the 157th to the 177th nucleotides of SEQ ID NO:
- 5, or (ii) the 157th to the 176th nucleotides of SEQ ID NO;
5, and wherein the antisense oligomer is a morpholino oligomer, or an oligonucleotide in which the sugar moiety and/or the phosphate-binding region of at least one nucleotide constituting the oligonucleotide is modified.
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Abstract
The present invention provides a pharmaceutical agent which causes skipping of the 55th, 45th, 50th or 44th exon in the human dystrophin gene with a high efficiency. The present invention provides an oligomer which efficiently enables to cause skipping of the 55th, 45th, 50th or 44th exon in the human dystrophin gene.
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9 Claims
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1. An antisense oligomer which causes skipping of the 55th exon in a human dystrophin gene, wherein the base sequence of the antisense oligomer consists of the base sequence of (i) the 157th to the 177th nucleotides of SEQ ID NO:
- 5, or (ii) the 157th to the 176th nucleotides of SEQ ID NO;
5, and wherein the antisense oligomer is a morpholino oligomer, or an oligonucleotide in which the sugar moiety and/or the phosphate-binding region of at least one nucleotide constituting the oligonucleotide is modified. - View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9)
- 5, or (ii) the 157th to the 176th nucleotides of SEQ ID NO;
Specification