Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded RNA
AssigneeDicerna Pharmaceuticals, Inc.
InventorsBob D. Brown, Henryk T. Dudek
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Abstract
This invention relates to compounds, compositions, and methods useful for reducing α-1 antitrypsin target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.
Claims22
A nucleic acid comprising an oligonucleotide strand consisting of 22 nucleotides in length, wherein said oligonucleotide strand has a region that is perfectly complementary to a contiguous sequence of α-1 antitrypsin mRNA consisting of 20 nucleotides in length, which contiguous sequence comprises a target α-1 antitrypsin mRNA sequence as set forth in SEQ ID NO:
- 1547, and wherein the nucleic acid comprises one or more modified nucleotides.
A double stranded nucleic acid (dsNA) comprising a sense strand and an antisense strand, wherein the antisense strand is 22 nucleotides in length, wherein the antisense strand has a region that is perfectly complementary to a contiguous sequence of α-1 antitrypsin mRNA consisting of 20 nucleotides in length, which contiguous sequence comprises a target α-1 antitrypsin mRNA sequence as set forth in SEQ ID NO:
- 1547, and wherein the dsNA comprises one or more modified nucleotides.
Dependencies are inferred from the claim text.