Single-stranded oligonucleotide
AssigneeNissan Chemical Corporation
InventorsYusuke Iriyama, Hiroyuki Nakajima, Tatsuro Kanaki
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AssigneeNissan Chemical Corporation
InventorsYusuke Iriyama, Hiroyuki Nakajima, Tatsuro Kanaki
Estimating the term…
Provided is a single-stranded oligonucleotide that is capable of controlling a target gene with high efficiency and can be easily produced. The single-stranded oligonucleotide is represented by the formula X-L-Y wherein X and Y hybridize by a first nucleotide sequence portion and a second nucleotide sequence portion. X is composed of 7 to 100 nucleotides, contains at least one modified nucleotide, and has a first nucleotide sequence that is capable of hybridizing with a second oligonucleotide and contains at least four contiguous nucleotides recognized by RNase H. Y is composed of 4 to 100 nucleotides, and has a second nucleotide sequence that is capable of hybridizing with a second oligonucleotide and contains at least one ribonucleotide. At least one of nucleotide sequence X and nucleotide sequence Y has an antisense sequence capable of hybridizing with a target RNA. L is a group derived from a third oligonucleotide that is degraded under physiological conditions.
A single-stranded oligonucleotide represented by the formula:
Dependencies are inferred from the claim text.
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Compounds having three or more nucleosides or nucleotides: nucleic acids or oligonucleotides having modified bases, i.e. other than adenine,…
Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming…: against enzymes
Carbohydrates; Sugars; Derivatives thereof: compounds having three or more nucleosides or nucleotides
Compounds having three or more nucleosides or nucleotides: nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or…
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This patent application is the U.S. national phase of International Patent Application No. PCT/JP2017/002831, filed Jan. 26, 2017, which claims the benefit of Japanese Patent Application No. 2016-012804, filed on Jan. 26, 2016, and Japanese Patent Application No. 2016-158833, filed on Aug. 12, 2016, which are incorporated by reference in their entireties herein.
Incorporated by reference in its entirety herein is a computer-readable nucleotide/amino acid sequence listing submitted concurrently herewith and identified as follows: 33,218 bytes ASCII (Text) file named “740008SequenceListing.txt,” created Jul. 25, 2018.
The present invention relates to a single-stranded oligonucleotide.
FIG. 1 is a conceptual diagram representing one aspect in which a first nucleotide sequence portion X2 and a second nucleotide sequence portion Y2 of an example of the present embodiment in the form of a single-stranded oligonucleotide hybridize within a molecule thereof.
FIG. 2 is a conceptual diagram representing one aspect in which a first nucleotide sequence portion X2 and a second nucleotide sequence portion Y2 of an example of the present embodiment in the form of a single-stranded oligonucleotide hybridize within a molecule thereof.
FIG. 3 is a conceptual diagram representing one aspect in which a first nucleotide sequence portion X2 and a second nucleotide sequence portion Y2 of an example of the present embodiment in the form of a single-stranded oligonucleotide hybridize within a molecule thereof.
The terms used in the present description are used in the sense in which they are ordinarily used in the art unless specifically indicated otherwise. The following provides an explanation of terms used in the present description. Furthermore, the terms used in the present description have the same meaning both in the case they are used alone and in the case they are used in conjunction with other terms unless specifically indicated otherwise.
“Antisense effect” refers to controlling the function of a target RNA by hybridizing a target RNA selected corresponding to a target gene and an oligonucleotide having a sequence complementary to a partial sequence thereof. For example, in the case the target RNA is mRNA, an antisense effect refers to translation of the aforementioned target RNA being suppressed by hybridization, an effect that converts a splicing function such as exon skipping, or the aforementioned target RNA being degraded as a result of recognition of a hybridized portion. Although examples of oligonucleotides in which the aforementioned antisense effect is demonstrated include DNA and oligodeoxyribonucleotides, oligonucleotides in which an antisense effect is demonstrated are not limited thereto, but rather may be RNA, oligoribonucleotides or oligonucleotides that have been designed to normally demonstrate an antisense function.
“Target RNA” refers to mRNA, mRNA precursor or ncRNA, and includes mRNA transcribed from genomic DNA encoding a target gene, mRNA not subjected to base modification, and mRNA precursor or ncRNA that have not been subjected to splicing. There are no particular limitations on “target RNA” for which the function thereof is controlled by an antisense effect, and examples thereof include RNA associated with genes for which expression increases in various diseases. “Target RNA” may be any RNA synthesized by DNA-dependent RNA polymerase, and is preferably mRNA or mRNA precursor. More preferably, the “target RNA” is mammal mRNA or mRNA precursor and even more preferably human mRNA or mRNA precursor.