Antisense nucleic acids
DC CAFCFirst Claim
Patent Images
1. A method of treating a DMD patient comprising intravenously administering to said patient an oligomer comprising:
- a) a phosphorodiamidate morpholino oligomer (PMO) 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, wherein the phosphorodiamidate morpholino monomers of said PMO have the formula;
1 Assignment
Litigations
1 Petition
Accused Products
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.
16 Citations
4 Claims
-
1. A method of treating a DMD patient comprising intravenously administering to said patient an oligomer comprising:
a) a phosphorodiamidate morpholino oligomer (PMO) 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, wherein the phosphorodiamidate morpholino monomers of said PMO have the formula;
-
2. A method of treating a DMD patient comprising intravenously administering to said patient an oligomer comprising:
a) a phosphorodiamidate morpholino oligomer (PMO) that is 100% complementary to the target sequence
-
3. A method of treating a DMD patient comprising intravenously administering to said patient a phosphorodiamidate morpholino oligomer (PMO) that causes skipping of the 53rd exon in a human dystrophin pre-mRNA, 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 said 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 morpholino oligomer hybridizes to said pre-mRNA with Watson-Crick base pairing, wherein each morpholino monomer has the formula;
- end of the 53rd exon in said human dystrophin pre-mRNA, wherein the 53rd exon in said human dystrophin pre-mRNA consists of a nucleotide sequence corresponding to SEQ ID NO;
-
4. A method of treating a DMD patient comprising intravenously administering to said patient a phosphorodiamidate morpholino oligomer (PMO) that causes skipping of the 53rd exon in a human dystrophin pre-mRNA, consisting of a 25-mer oligomer that is 100% complementary to the target pre-mRNA sequence
Specification