Optimized sirna scaffolds
AssigneeUniversity of Massachusetts
InventorsAnastasia Khvorova, Daniel O'Reilly, Vignesh Narayan Hariharan, Clemens Lochmann, David Cooper
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Abstract
This disclosure relates to novel modified oligonucleotides with increased stability and extended in vivo mRNA silencing activity.
Claims31
An RNA molecule comprising a 5′ end and a 3′ end, wherein the RNA molecule comprises at least one alkyl modification within nucleotide positions 1-5 of one or both of the 5′ end and 3′ end.
5-6. (canceled)
8-14. (canceled)
A double stranded (ds) RNA, comprising an antisense strand with a 5′ end and a 3′ end, and a sense strand with a 5′ end and a 3′ end, wherein the antisense strand comprises at least one alkyl modification.
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26-27. (canceled)
29-33. (canceled)
A double stranded (ds) RNA, comprising an antisense strand and a sense strand, each strand with a 5′ end and a 3′ end, and at least one single stranded nucleotide overhang of 2-5 nucleotides, wherein the single stranded nucleotide overhang comprises at least two nucleotide modifications selected from the group consisting of a 2′-deoxy modification, a 2′-MOE modification, an LNA modification, a UNA modification, and an alkyl modification.
38-39. (canceled)
A double stranded (ds) RNA, comprising an antisense strand and a sense strand, each strand with a 5′ end and a 3′ end, wherein the antisense strand or sense strand comprises a chemical modification pattern of any one of the chemical modification patterns provided in Tables 1-8.
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Dependencies are inferred from the claim text.