Single-stranded nucleic acid molecule for regulating expression of gene having delivering function
First Claim
1. An isolated molecule for inhibiting expression of a target gene, wherein the molecule has a delivery function and comprises, in the following order, a 5′
- -side region (Xc), a linker region (Lx), an inner region (Z), a linker region (Ly) and a 3′
-side region (Yc) which regions are linked together, whereinthe inner region (Z) is constituted by linkage of an inner 5′
-side region (X) and an inner 3′
-side region (Y),the 5′
-side region (Xc), the inner 5′
-side region (X), the inner 3′
-side region (Y) and the 3′
side region (Yc) are each composed of a nucleotide residue or a nucleotide sequence,the 5′
-side region (Xc) is complementary to the inner 5′
-side region (X),the 3′
-side region (Yc) is complementary to the inner 3′
-side region (Y),at least one of the inner region (Z), the 5′
-side region (Xc) and the 3′
-side region (Yc) comprises an expression inhibitory sequence that inhibits expression of a target gene,the linker region (Lx) is linked to the 3′
-end of the 5′
-side region (Xc) and the 5′
-end of the inner 5′
-side region (X),the linker region (Ly) is linked to the 3′
-end of the inner 3′
-side region (Y) and the 5′
-end of the 3′
-side region (Yc),the linker region (Lx) and the linker region (Ly) are each composed of a nucleotide or non-nucleotide residue or a sequence of at least one nucleotide and non-nucleotide residues,wherein at least one selected from the group consisting of the linker region (Lx) and the linker region (Ly) comprises a lysine residue in such a manner that (i) the 5′
-side region (Xc) and the inner 5′
-side region (X) or (ii) the inner 3′
-side region (Y) and the 3′
-side region (Yc) are linked together via the lysine residue, anda lipid is covalently bound to the molecule via the lysine residue.
1 Assignment
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Accused Products
Abstract
The invention provides a single-stranded nucleic acid capable of inhibiting expression of a target gene having a delivery function. The nucleic acid contains, from the 5′-side to the 3′-side, a 5′-side region (Xc), a linker region (Lx), an inner region (Z), a linker region (Ly) and a 3′-side region (Yc) in this order, wherein the inner region (Z) is constituted by linkage of the inner 5′-side region (X) and the inner 3′-side region (Y), the 5′-side region (Xc) is complementary to the inner 5′-side region (X), the 3′-side region (Yc) is complementary to the inner 3′-side region (Y), at least one of the inner region (Z), the 5′-side region (Xc) and the 3′-side region (Yc) comprises an expression inhibitory sequence that inhibits expression of a target gene, and at least one of the 5′-terminus, the 3′-terminus, the linker region (Lx) and the linker region (Ly) is bound to a bio-related substance.
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Citations
20 Claims
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1. An isolated molecule for inhibiting expression of a target gene, wherein the molecule has a delivery function and comprises, in the following order, a 5′
- -side region (Xc), a linker region (Lx), an inner region (Z), a linker region (Ly) and a 3′
-side region (Yc) which regions are linked together, whereinthe inner region (Z) is constituted by linkage of an inner 5′
-side region (X) and an inner 3′
-side region (Y),the 5′
-side region (Xc), the inner 5′
-side region (X), the inner 3′
-side region (Y) and the 3′
side region (Yc) are each composed of a nucleotide residue or a nucleotide sequence,the 5′
-side region (Xc) is complementary to the inner 5′
-side region (X),the 3′
-side region (Yc) is complementary to the inner 3′
-side region (Y),at least one of the inner region (Z), the 5′
-side region (Xc) and the 3′
-side region (Yc) comprises an expression inhibitory sequence that inhibits expression of a target gene,the linker region (Lx) is linked to the 3′
-end of the 5′
-side region (Xc) and the 5′
-end of the inner 5′
-side region (X),the linker region (Ly) is linked to the 3′
-end of the inner 3′
-side region (Y) and the 5′
-end of the 3′
-side region (Yc),the linker region (Lx) and the linker region (Ly) are each composed of a nucleotide or non-nucleotide residue or a sequence of at least one nucleotide and non-nucleotide residues, wherein at least one selected from the group consisting of the linker region (Lx) and the linker region (Ly) comprises a lysine residue in such a manner that (i) the 5′
-side region (Xc) and the inner 5′
-side region (X) or (ii) the inner 3′
-side region (Y) and the 3′
-side region (Yc) are linked together via the lysine residue, anda lipid is covalently bound to the molecule via the lysine residue. - View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20)
- -side region (Xc), a linker region (Lx), an inner region (Z), a linker region (Ly) and a 3′
Specification