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US 2026/0250686 A1Published application
Sirna structures for high activity and reduced off target
AssigneeNitto Denko Corporation
InventorsKenjirou Minomi, Jens Harborth, Cima Cina, Wenbin Ying, Jane Zheng, Narendra Vaish
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Abstract
Compounds, compositions, and methods for modulating expression of target genes using RNA interference. RNAi structures and molecules can be used for modulating or silencing the expression of genes, with high levels of RNAi activity and reduced off target actions. Advantageous structures include siRNAs targeted to any gene having one or more 2′-deoxy nucleotides located in the seed region. The RNA interference molecules can be used in methods for preventing or treating diseases.
Claims20
A nucleic acid molecule, wherein:
- a) the molecule has a polynucleotide sense strand and a polynucleotide antisense strand;
- b) each strand of the molecule is from 15 to 30 nucleotides in length;
- c) a contiguous region of from 15 to 30 nucleotides of the antisense strand is complementary to a sequence of a mRNA;
- d) at least a portion of the sense strand is complementary to at least a portion of the antisense strand, and the molecule has a duplex region of from 15 to 30 nucleotides in length, wherein one or more of the nucleotides in the duplex region at positions 3 to 8 from the 5′ end of the antisense strand are deoxynucleotides, and all the remaining nucleotides in the duplex region of the antisense strand are not deoxynucleotides;
- and
- wherein the molecule is modified or chemically-modified at least at the 5′ end or the 3′ end of the antisense strand.
3 more elements
Dependencies are inferred from the claim text.