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US 10,968,452 B2Granted
Polynucleotide agents targeting aminolevulinic acid synthase-1 (ALAS1) and uses thereof
AssigneeAlnylam Pharmaceuticals, Inc.
InventorGregory Hinkle
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Abstract
The invention relates to polynucleotide agents, e.g., antisense polunucleotide agents, targeting the ALAS1 gene, and methods of using such agents to alter (e.g., inhibit) expression of ALAS1 and to treat ALAS1 associated diseases, e.g., porphyria.
Claims13
A single-stranded antisense polynucleotide agent for inhibiting aminolevulinic acid synthase-1 (ALAS1) expression, comprising the nucleotide sequence 5′-UCAUGGGCCACAUCACACAG-3′ (SEQ ID NO:730), wherein the agent is 20-40 nucleotides in length, wherein the polynucleotide comprises a gap segment consisting of linked 8 to 14 deoxynucleotides;
- a 5′-wing segment consisting of 1 to 6 linked nucleotides;
- and a 3′-wing segment consisting of 1 to 6 linked nucleotides;
- wherein the gap segment is positioned between the 5′-wing segment and the 3′-wing segment and wherein each nucleotide of each wing segment comprises a modified sugar.
Dependencies are inferred from the claim text.