Abstract: An object of the invention is to provide a low toxicity antisense nucleic acid medicine that can modulate expression of a target transcriptional product in the central nervous system and other sites of a subject. Provided is a low toxicity composition for modulating expression of a target transcriptional product in a site such as the central nervous system of a subject, having a nucleic acid complex formed by annealing together a first nucleic acid strand having an antisense oligonucleotide region with respect to the target transcriptional product, and a second nucleic acid strand having a complementary region that is complementary to at least part of the first nucleic acid strand.

Abstract: Provided is a nucleic acid inhibiting a function of a target miRNA. Provided is a double-stranded nucleic acid complex comprising a first nucleic acid strand of 6 to 30 nucleotide length that hybridizes to a target miRNA to inhibit a function of the target miRNA, and a second nucleic acid strand complementary to the first nucleic acid strand, wherein the first nucleic acid strand is a mixmer comprising a natural nucleoside and a non-natural nucleoside, and the second nucleic acid strand comprises at least one of one or more modified internucleoside linkages and one or more sugar modified nucleosides.

Abstract: Provided is a nucleic acid strand that can efficiently deliver an antisense oligonucleotide into the body, particularly a nucleic acid complex comprising a first nucleic acid strand and a second nucleic acid strand, wherein the first nucleic acid strand includes a base sequence that is capable of hybridizing with at least a portion of a target transcription product, and exerts an antisense effect on the target transcription product; the second nucleic acid strand includes a complementary region having a base sequence complementary to the first nucleic acid strand and at least one overhang region located on the 5? and/or 3? side of the complementary region; and the first nucleic acid strand is annealed to the complementary region in the second nucleic acid strand.

Abstract: Disclosed are double-stranded nucleic acid agents that can deliver a therapeutic oligonucleotide within a biological sample, and methods for using the same. In one embodiment, the double-stranded nucleic acid agent comprises a first strand comprising a first RNA region, and a second strand comprising a first DNA region, wherein said first RNA region and said first DNA region are hybridized as a RNA/DNA heteroduplex. Said first strand further comprises a nucleic acid therapeutic oligonucleotide region that is capable of being cleaved from at least one nucleotide in said first RNA region. Methods for using the double-stranded nucleic acid agents include methods for delivering the therapeutic oligonucleotide as a single strand by cleaving it from at least a portion of the first RNA region. The methods further include delivering the double-stranded nucleic acid agent and thus the therapeutic oligonucleotide to a target site within the body of a treatment subject.

Abstract: An object of the invention is to provide a low toxicity antisense nucleic acid medicine that can modulate expression of a target transcriptional product in the central nervous system and other sites of a subject. Provided is a low toxicity composition for modulating expression of a target transcriptional product in a site such as the central nervous system of a subject, having a nucleic acid complex formed by annealing together a first nucleic acid strand having an antisense oligonucleotide region with respect to the target transcriptional product, and a second nucleic acid strand having a complementary region that is complementary to at least part of the first nucleic acid strand.

Abstract: Problem to be Solved It is intended to provide an industrially preferable fluoroalkylating agent and use thereof. Solution The present invention provides a fluoroalkylating agent represented by the general formula (1) wherein R1 is a C1 to C8 fluoroalkyl group; R2 and R3 are each independently a C1 to C12 alkyl group or the like; Y1 to Y4 are each independently a hydrogen atom, a halogen atom, or the like; and X? is a monovalent anion. A compound of the general formula (3): R4—S—R1 having an introduced C1 to C8 fluoroalkyl group is easily obtained by reacting a compound of the general formula (2): R4—S—Z wherein R4 is a hydrocarbon group or the like; and Z is a leaving group, with the compound of the general formula (1).

Abstract: Provided is a nucleic acid inhibiting a function of a target miRNA. Provided is a double-stranded nucleic acid complex comprising a first nucleic acid strand of 6 to 30 nucleotide length that hybridizes to a target miRNA to inhibit a function of the target miRNA, and a second nucleic acid strand complementary to the first nucleic acid strand, wherein the first nucleic acid strand is a mixmer comprising a natural nucleoside and a non-natural nucleoside, and the second nucleic acid strand comprises at least one of one or more modified internucleoside linkages and one or more sugar modified nucleosides.

Abstract: Provided is a nucleic acid strand that can efficiently deliver an antisense oligonucleotide into the body, particularly a nucleic acid complex comprising a first nucleic acid strand and a second nucleic acid strand, wherein the first nucleic acid strand includes a base sequence that is capable of hybridizing with at least a portion of a target transcription product, and exerts an antisense effect on the target transcription product; the second nucleic acid strand includes a complementary region having a base sequence complementary to the first nucleic acid strand and at least one overhang region located on the 5? and/or 3? side of the complementary region; and the first nucleic acid strand is annealed to the complementary region in the second nucleic acid strand.

Abstract: Problem to be Solved It is intended to provide an industrially preferable fluoroalkylating agent and use thereof. Solution The present invention provides a fluoroalkylating agent represented by the general formula (1) wherein R1 is a C1 to C8 fluoroalkyl group; R2 and R3 are each independently a C1 to C12 alkyl group or the like; Y1 to Y4 are each independently a hydrogen atom, a halogen atom, or the like; and X? is a monovalent anion. A compound of the general formula (3): R4—S—R1 having an introduced C1 to C8 fluoroalkyl group is easily obtained by reacting a compound of the general formula (2): R4—S—Z wherein R4 is a hydrocarbon group or the like; and Z is a leaving group, with the compound of the general formula (1).

Abstract: Problem to be Solved It is intended to provide an industrially preferable fluoroalkylating agent and use thereof. Solution The present invention provides a fluoroalkylating agent represented by the general formula (1) wherein R1 is a C1 to C8 fluoroalkyl group; R2 and R3 are each independently a C1 to C12 alkyl group or the like; Y1 to Y4 are each independently a hydrogen atom, a halogen atom, or the like; and X? is a monovalent anion. A compound of the general formula (3): R4—S—R1 having an introduced C1 to C8 fluoroalkyl group is easily obtained by reacting a compound of the general formula (2): R4—S—Z wherein R4 is a hydrocarbon group or the like; and Z is a leaving group, with the compound of the general formula (1).

Abstract: Disclosed are double-stranded antisense nucleic acid complexes that can efficiently alter the processing of RNA in a cell via an antisense effect, and methods for using the same. One method comprises contacting with the cell a double-stranded nucleic acid complex comprising: a first nucleic acid strand annealed to a second nucleic acid strand, wherein: the first nucleic acid strand comprises (i) nucleotides independently selected from natural DNA nucleotides, modified DNA nucleotides, and nucleotide analogs, (ii) no regions that have 4 or more consecutive natural DNA nucleotides, (iii) the total number of natural DNA nucleotides, modified DNA nucleotides, and nucleotide analogs in the first nucleic acid strand is from 8 to 100, and (iv) the first nucleic acid strand is capable of hybridizing to RNA inside of the cell; and the second nucleic acid strand comprises nucleotides independently selected from natural RNA nucleotides, modified RNA nucleotides, and nucleotide analogs.

Abstract: [Problem to be Solved ] Provided is a nucleic acid complex, preferably a double-stranded nucleic acid complex, having an excellent effect of suppressing the expression of a target gene. [Solution] The problem is solved by using a nucleic acid complex, preferably a double-stranded nucleic acid complex, comprising an active moiety comprising an antisense nucleic acid complementary to a transcript, for example, a transcript of a target gene, and a carrier moiety comprising a nucleic acid complementary to the nucleic acid, and having at least one bulge structure.

Abstract: Provided is a nucleic acid complex, preferably a double-stranded nucleic acid complex, having an excellent effect of suppressing the expression of a target gene. Further provided is a nucleic acid complex, that is, a double-stranded nucleic acid complex, comprising an active moiety comprising an antisense nucleic acid complementary to a transcript, for example, a transcript of a target gene, and a carrier moiety comprising a nucleic acid comprising DNA, preferably a DNA-based nucleic acid, which is complementary to the above-described nucleic acid.

Abstract: Problem to be Solved It is intended to provide an industrially preferable fluoroalkylating agent and use thereof. Solution The present invention provides a fluoroalkylating agent represented by the general formula (1) wherein R1 is a C1 to C8 fluoroalkyl group; R2 and R3 are each independently a C1 to C12 alkyl group or the like; Y1 to Y4 are each independently a hydrogen atom, a halogen atom, or the like; and X? is a monovalent anion. A compound of the general formula (3): R4—S—R1 having an introduced C1 to C8 fluoroalkyl group is easily obtained by reacting a compound of the general formula (2): R4—S—Z wherein R4 is a hydrocarbon group or the like; and Z is a leaving group, with the compound of the general formula (1).

Abstract: Disclosed are double-stranded nucleic acid agents that can deliver a therapeutic oligonucleotide within a biological sample, and methods for using the same. In one embodiment, the double-stranded nucleic acid agent comprises a first strand comprising a first RNA region, and a second strand comprising a first DNA region, wherein said first RNA region and said first DNA region are hybridized as a RNA/DNA heteroduplex. Said first strand further comprises a nucleic acid therapeutic oligonucleotide region that is capable of being cleaved from at least one nucleotide in said first RNA region. Methods for using the double-stranded nucleic acid agents include methods for delivering the therapeutic oligonucleotide as a single strand by cleaving it from at least a portion of the first RNA region. The methods further include delivering the double-stranded nucleic acid agent and thus the therapeutic oligonucleotide to a target site within the body of a treatment subject.

Abstract: Disclosed are double-stranded antisense nucleic acid complexes that can efficiently alter the processing of RNA in a cell via an antisense effect, and methods for using the same. One method comprises contacting with the cell a double-stranded nucleic acid complex comprising: a first nucleic acid strand annealed to a second nucleic acid strand, wherein: the first nucleic acid strand comprises (i) nucleotides independently selected from natural DNA nucleotides, modified DNA nucleotides, and nucleotide analogs, (ii) no regions that have 4 or more consecutive natural DNA nucleotides, (iii) the total number of natural DNA nucleotides, modified DNA nucleotides, and nucleotide analogs in the first nucleic acid strand is from 8 to 100, and (iv) the first nucleic acid strand is capable of hybridizing to RNA inside of the cell; and the second nucleic acid strand comprises nucleotides independently selected from natural RNA nucleotides, modified RNA nucleotides, and nucleotide analogs.