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US 11,697,812 B2Granted
Methods and compositions for the specific inhibition of transthyretin (TTR) by double-stranded RNA
AssigneeDicerna Pharmaceuticals, Inc.
InventorsBob D. Brown, Henryk T. Dudek
Life of this patent
Estimating the term…
Abstract
This invention relates to compounds, compositions, and methods useful for reducing transthyretin (TTR) target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.
Claims20
A double stranded nucleic acid (dsNA) comprising a first oligonucleotide and a second oligonucleotide, wherein the first oligonucleotide and the second oligonucleotide form a duplex region, wherein the dsNA comprises a modified nucleotide, and wherein the dsNA sequences are selected from:
- TTR-156 Target:
- first oligonucleotide:
- (SEQ ID NO:
- 1194)
- 5’-GCAGGAUGGCUUCUCAUCGUCUGCU-3’
- second oligonucleotide:
- (SEQ ID NO:
- 426)
- 3’ -ACCGUCCUACCGAAGAGUAGCAGACGA-5’;
- TTR-242 Target:
- first oligonucleotide:
- (SEQ ID NO:
- 1211)
- 5’ -UCCAAGUGUCCUCUGAUGGUCAAAG-3’
- second oligonucleotide:
- (SEQ ID NO:
- 443)
- 3’-UUAGGUUCACAGGAGACUACCAGUUUC-5’;
- TTR-244 Target:
- first oligonucleotide:
- (SEQ ID NO:
- 1213)
- 5’-CAAGUGUCCUCUGAUGGUCAAAGUU-3’
- second oligonucleotide:
- (SEQ ID NO:
- 445)
- 3’ -AGGUUCACAGGAGACUACCAGUUUCAA-5’;
- and
- TTR-255 Target:
- first oligonucleotide:
- (SEQ ID NO:
- 1224)
- 5’-UGAUGGUCAAAGUUCUAGAUGCUGU-3’
- second oligonucleotide:
- (SEQ ID NO:
- 456)
- 3’-AGACUACCAGUUUCAAGAUCUACGACA-5’.
34 more elements
Dependencies are inferred from the claim text.