Figure 2.

Antisense Oligonucleotides can be conjugated to antibodies targeting the Transferrin Receptor 1 (TfR1) and penetrate cells by receptor-mediated endocytosis. (A) Schematic design of an antibody–ASO conjugate. The ASO is conjugated directly to the antibody using covalent unions. (B) Antibodies targeting TfR1, a highly expressed cell-surface receptor of DM1 in target tissues such as skeletal and cardiac muscles, have been used to deliver antisense cargo. The AOC enters the cell by receptor-mediated endocytosis resulting on an enhanced ASO delivery. The affinity of TfR1antibodies can be fine-tuned to maximise the interaction with receptors in the BBB allowing for the compound to be delivered to the CNS (in mice) after systemic administration [85].