Antisense nucleic acids
AssigneesNippon Shinyaku Co., Ltd., National Center of Neurology and Psychiatry
InventorsNaoki Watanabe, Youhei Satou, Shin'ichi Takeda, Tetsuya Nagata
Life of this patent
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Abstract
The present invention provides an oligomer which efficiently enables to cause skipping of the 53rd exon in the human dystrophin gene. Also provided is a pharmaceutical composition which causes skipping of the 53rd exon in the human dystrophin gene with a high efficiency.
Claims12
A method comprising administering to a patient with DMD an antisense phosphorodiamidate morpholino oligomer (PMO) consisting of a 25-mer oligomer that is 100% complementary to the 36th to the 60th nucleotides from the 5′ end of the 53rd exon in a human dystrophin pre-mRNA, wherein the 53rd exon in said human dystrophin pre-mRNA consists of a nucleotide sequence corresponding to SEQ ID NO:
- 1, wherein said PMO hybridizes to said human dystrophin pre-mRNA with Watson-Crick base pairing, and wherein skipping of the 53rd exon is induced in said patient.
A method of inducing exon 53 skipping in a patient with DMD comprising administering to said patient an antisense phosphorodiamidate morpholino oligomer (PMO) consisting of a 25-mer oligomer that is 100% complementary to the 36th to the 60th nucleotides from the 5′ end of the 53rd exon in a human dystrophin pre-mRNA, wherein the 53rd exon in said human dystrophin pre-mRNA consists of a nucleotide sequence corresponding to SEQ ID NO:
- 1, and wherein said PMO hybridizes to said human dystrophin pre-mRNA with Watson-Crick base pairing.
A method comprising administering to a patient with DMD an antisense phosphorodiamidate morpholino oligomer (PMO) consisting of a 25-mer oligomer that is 100% complementary to the target sequence 5′-GAACACCUUCAGAACCGGAGGCAAC-3′ (SEQ ID NO:
- 124), wherein the 53rd exon in a human dystrophin pre-mRNA consists of a nucleotide sequence corresponding to SEQ ID NO:
- 1, wherein said PMO hybridizes to said human dystrophin pre-mRNA with Watson-Crick base pairing, and wherein skipping of the 53rd exon is induced in said patient.
A method of inducing exon 53 skipping in a patient with DMD comprising administering to said patient an antisense phosphorodiamidate morpholino oligomer (PMO) consisting of a 25-mer oligomer that is 100% complementary to the target sequence 5′-GAACACCUUCAGAACCGGAGGCAAC-3′ (SEQ ID NO:
- 124), wherein the 53rd exon in a human dystrophin pre-mRNA consists of a nucleotide sequence corresponding to SEQ ID NO:
- 1, and wherein said PMO hybridizes to said human dystrophin pre-mRNA with Watson-Crick base pairing.
Dependencies are inferred from the claim text.