Targeted delivery of therapeutic oligonucleotides through conjugation to a homing ligand enables modulation of disease targets in specific cell types that are intractable to small molecules and antibodies.
The homing ligand can a small molecule, peptide, carbohydrate or aptamer. The linker chemistry is designed so that the conjugate cleavage occurs in the desired cell type to deliver the drug where it can modulate the disease-causing target. The linker design is also critical to limiting the risk of toxicity driven by the linker fragments that may be released upon conjugate cleavage or degradation.
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