Rna silencing agents and methods of use
AssigneeADARx Pharmaceuticals, Inc.
InventorsZhen Li, Zhiqing (Joel) Zhou
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Abstract
Aspects of the disclosure provide nucleic acids for reducing expression of a target RNA. In some aspects, the disclosure provides nucleic acid modifications and base-pairing configurations useful in the design of nucleic acids for RNA interference.
Claims54
A nucleic acid for reducing expression of a target mRNA, the nucleic acid comprising an antisense strand of 15 to 31 nucleotides in length having a sequence that is at least 90% complementary to a contiguous sequence of the target mRNA, wherein the sequence of the antisense strand comprises, at position 14 from its 5′ end, an abasic site or a nucleotide that does not form a canonical base pair with a target nucleotide at a corresponding position on the contiguous sequence of the target mRNA.
A nucleic acid for reducing expression of a target mRNA, the nucleic acid comprising an antisense strand of Formula (I):
- 5′-X01X02X03X04X05X06X07X08X09X10X11X12X13X14(XY)bNa-3′ (I),
- wherein:
- each instance of N and XY is independently any type of nucleotide;
- a is an integer from 0-2, inclusive;
- b is an integer from 1-17, inclusive;
- X01-X13 are each independently any type of nucleotide, with the proviso that X01—(XY)b is at least 90% complementary to a contiguous nucleotide sequence of the target mRNA;
- and
- X14 is an abasic site or a nucleotide that does not form a canonical base pair with a target nucleotide at a corresponding position on the contiguous nucleotide sequence of the target mRNA.
A composition comprising a nucleic acid of any one of the preceding claims and a counterion.
A composition comprising a nucleic acid of any one of the preceding claims and a pharmaceutically acceptable carrier.
Dependencies are inferred from the claim text.