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AssigneeAlnylam Pharmaceuticals, Inc.
InventorsMark K. SCHLEGEL, Maja JANAS, Vasant R. JADHAV, Muthiah MANOHARAN, Kallanthottathil G. RAJEEV, Muthusamy JAYARAMAN, Alexander V. KEL'IN, Shigeo MATSUDA, Klaus CHARISSE, Jayaprakash K. NAIR, Martin A. MAIER, Alfica SEHGAL, Christopher BROWN, Christopher THEILE, Stuart MILSTEIN
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One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene. The antisense strand of the dsRNA molecule comprises at least one thermally destabilizing nucleotide occurring at a seed region; the dsRNA comprises at least four 2′-fluoro modifications, and the sense strand of the dsRNA molecule comprises ligand, wherein the ligand is an ASGPR ligand. Other aspects of the invention relate to pharmaceutical compositions comprising these dsRNA molecules suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA molecules, e.g., for the treatment of various disease conditions.
-27. (canceled)
A double-stranded RNA (dsRNA) capable of inhibiting the expression of a target gene, comprising a sense strand and an antisense strand, each strand having 14 to 40 nucleotides, wherein the dsRNA comprises a duplex region of 12-40 nucleotide pairs in length, and wherein the antisense strand has sufficient complementarity to the target sequence to mediate RNA interference;
Dependencies are inferred from the claim text.