TARGETED INTRACELLULAR DELIVERY OF OLIGONUCLEOTIDES VIA CONJUGATION WITH SMALL MOLECULE LIGANDS

摘要

An oligonucleotide covalently coupled to at least one ligand for a membrane bound protein is provided. The ligand is preferably so coupled through a linking group. The linking group preferably comprising a phosphate group covalently coupled to a substituted or unsubstituted aliphatic group or a substituted or unsubstituted aliphatic oxide group. The compounds may be provided as salts, including pharmaceutically acceptable salts, thereof. Methods of making and using the same are also provided.