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
Estimating the term…
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.
Claims6
1-14. (canceled)
A composition comprising a cationic polymer with a phosphorodiamidate morpholino oligomer (PMO) antisense oligomer that is 100% complementary to a target sequence of 5′-GAACACCUUCAGAACCGGAGGCAAC-3′ (SEQ ID NO:
- 124),
- wherein said PMO antisense oligomer causes skipping of the 53rd exon in a human dystrophin pre-mRNA,
- wherein said PMO antisense oligomer hybridizes to said target sequence with Watson-Crick base pairing under physiological conditions,
- wherein said PMO antisense oligomer has exactly 25 phosphorodiamidate morpholino monomers, each of which has the formula:
- wherein each of R2 and R3 represents a methyl, wherein Base is a nucleobase selected from the group consisting of uracil, cytosine, thymine, adenine, and guanine, and
- wherein the 5′ end of said PMO antisense oligomer has a formula selected from the group consisting of:
A composition that causes skipping of the 53rd exon in a human dystrophin pre-mRNA, comprising a cationic carrier with a 25-mer phosphorodiamidate morpholino oligomer (PMO) antisense oligomer,
- wherein said PMO antisense oligomer is 100% complementary to a target sequence of 5′-GAACACCUUCAGAACCGGAGGCAAC-3′ (SEQ ID NO:
- 124),
- wherein each phosphorodiamidate morpholino monomer of said PMO antisense oligomer has the formula:
- wherein each of R2 and R3 represents a methyl, wherein Base is a nucleobase selected from the group consisting of uracil, cytosine, thymine, adenine, and guanine, and
- wherein said cationic carrier promotes delivery of said composition to a muscle tissue.
A composition comprising:
- a) a 25-mer phosphorodiamidate morpholino oligomer (PMO) antisense oligomer that is 100% complementary to a target sequence of 5′-GAACACCUUCAGAACCGGAGGCAAC-3′ (SEQ ID NO:
- 124), wherein said PMO antisense oligomer causes skipping of the 53rd exon in a human dystrophin pre-mRNA;
- and
- b) a carrier to promote delivery of said PMO antisense oligomer to a muscle tissue.
Dependencies are inferred from the claim text.