CPC A61K 47/6807 (2017.08) [A61K 31/712 (2013.01); A61K 31/713 (2013.01); A61K 39/395 (2013.01); A61K 47/6849 (2017.08); A61P 21/00 (2018.01); C07K 16/18 (2013.01); C12N 15/113 (2013.01); A61K 9/5107 (2013.01); C12N 2310/14 (2013.01); C12N 2310/315 (2013.01); C12N 2310/317 (2013.01); C12N 2310/3513 (2013.01); C12N 2310/3515 (2013.01); C12N 2320/31 (2013.01); C12N 2320/32 (2013.01)] | 13 Claims |
1. A small interfering RNA (siRNA)-antibody conjugate comprising an anti-transferrin receptor antibody or antigen-binding fragment thereof conjugated to an siRNA that hybridizes to a target sequence of human Atrogin-1 mRNA and mediates RNA interference against the human Atrogin-1 mRNA preferentially in a muscle cell, wherein the siRNA comprises a guide strand and a passenger strand, and the guide strand comprises a sequence selected from SEQ ID NO: 318, SEQ ID NO: 321, SEQ ID NO: 325, SEQ ID NO: 327, SEQ ID NO:333, SEQ ID NO: 337, SEQ ID NO:339, SEQ ID NO:342, SEQ ID NO:344, SEQ ID NO: 346, SEQ ID NO:348, SEQ ID NO:350, SEQ ID NO:353, SEQ ID NO:358, SEQ ID NO:359, SEQ ID NO:361, SEQ ID NO:363, SEQ ID NO:365, SEQ ID NO:366 and SEQ ID NO: 368.
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