Antisense nucleic acids
First Claim
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1. An antisense oligomer which causes skipping of the 50th exon in a human dystrophin gene, consisting of a nucleotide sequence complementary to any one of the nucleotide sequences consisting of the 106th to the 126th, the 107th to the 127th, the 108th to the 127th, the 108th to the 128th, or the 109th to the 129th nucleotides, from the 5′
- end of the human dystrophin gene'"'"'s 50th exon, wherein the 50exon of the human dystrophin gene consists of position 1 to 109 of the nucleotide sequence SEQ ID NO;
3, and wherein the antisense oligomer is a morpholino opligomer 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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Citations
9 Claims
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1. An antisense oligomer which causes skipping of the 50th exon in a human dystrophin gene, consisting of a nucleotide sequence complementary to any one of the nucleotide sequences consisting of the 106th to the 126th, the 107th to the 127th, the 108th to the 127th, the 108th to the 128th, or the 109th to the 129th nucleotides, from the 5′
- end of the human dystrophin gene'"'"'s 50th exon, wherein the 50exon of the human dystrophin gene consists of position 1 to 109 of the nucleotide sequence SEQ ID NO;
3, and wherein the antisense oligomer is a morpholino opligomer 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)
- end of the human dystrophin gene'"'"'s 50th exon, wherein the 50exon of the human dystrophin gene consists of position 1 to 109 of the nucleotide sequence SEQ ID NO;
Specification