ANTISENSE NUCLEIC ACIDS
First Claim
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1. An antisense oligomer which causes skipping of the 55th exon in the human dystrophin gene, consisting of a nucleotide sequence complementary to any one of the nucleotide sequences consisting of the 14th to the 34th or the 15th to the 34th nucleotides, from the 5′
- end of the 55th exon in the human dystrophin gene, wherein the antisense oligomer is a morpholino oligomer, or an oligonucleotide 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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22 Claims
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1. An antisense oligomer which causes skipping of the 55th exon in the human dystrophin gene, consisting of a nucleotide sequence complementary to any one of the nucleotide sequences consisting of the 14th to the 34th or the 15th to the 34th nucleotides, from the 5′
- end of the 55th exon in the human dystrophin gene, wherein the antisense oligomer is a morpholino oligomer, or an oligonucleotide which the sugar moiety and/or the phosphate-binding region of at least one nucleotide constituting the oligonucleotide is modified.
- View Dependent Claims (6, 15, 16, 17, 18, 19, 20, 21, 22)
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2-5. -5. (canceled)
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7-14. -14. (canceled)
Specification