CPC A61K 47/6803 (2017.08) [A61K 31/7088 (2013.01); A61K 31/713 (2013.01); A61K 47/60 (2017.08); A61K 47/6455 (2017.08); A61K 47/6807 (2017.08); A61K 47/6849 (2017.08); A61K 48/0058 (2013.01); A61K 48/0066 (2013.01); A61K 48/0083 (2013.01); A61P 21/00 (2018.01); C07K 14/003 (2013.01); C07K 16/2881 (2013.01); C07K 16/40 (2013.01); C12N 15/113 (2013.01); A61K 38/00 (2013.01); C12N 2310/11 (2013.01); C12N 2310/315 (2013.01); C12N 2310/3233 (2013.01); C12N 2310/3513 (2013.01); C12N 2320/32 (2013.01); C12N 2320/33 (2013.01)] | 24 Claims |
1. A method of treating muscular dystrophy in a subject in need thereof, comprising:
administering to the subject a single stranded oligonucleotide conjugate comprising an anti-transferrin receptor antibody or antigen binding fragment thereof conjugated to a single stranded oligonucleotide hybridizing to an acceptor splice site, a donor splice site, or an exonic splice enhancer element of a pre-mRNA transcript of the DMD gene;
wherein the single stranded oligonucleotide induces exon skipping in said pre-mRNA transcript to generate an mRNA transcript encoding a truncated dystrophin protein, thereby treating the muscular dystrophy in the subject.
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