CPC C12N 15/113 (2013.01) [A61K 31/7125 (2013.01); A61K 47/6807 (2017.08); A61K 47/6849 (2017.08); A61K 47/6889 (2017.08); A61K 48/005 (2013.01); A61P 21/00 (2018.01); A61P 21/06 (2018.01); C07K 16/2881 (2013.01); C07K 19/00 (2013.01); C07K 2317/55 (2013.01); C12N 2310/11 (2013.01); C12N 2310/14 (2013.01); C12N 2310/31 (2013.01); C12N 2310/313 (2013.01); C12N 2310/321 (2013.01); C12N 2310/3231 (2013.01); C12N 2310/3513 (2013.01); C12N 2320/32 (2013.01)] | 16 Claims |
1. A polynucleotide-antibody conjugate comprising an anti-transferrin receptor antibody or antigen binding fragment thereof conjugated to a polynucleotide, wherein the polynucleotide is from 19 to 30 nucleotides in length, wherein the polynucleotide comprises a nucleic acid sequence selected from a group consisting of SEQ ID NOs: 72, 76, 126, 131, 132, 134, 135, 136, 212, 216, 266, 271, 272, 274, 275, and 276 and wherein the polynucleotide mediates RNA interference against DUX4 mRNA and downregulation of DUX4 biomarker mRNA level.
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