Gene transfer to muscle cells mediated by receptor mannose 6-phosphate.

摘要

Use of a conjugate of a glycoside linked to an oligonucleotide selected from RNA, DNA, morpholino, 2 '' - O-methyl RNA, 2 '' - O-allyl RNA, peptide nucleic acid (PNA) and locked nucleic acid (LNA) , wherein said glycoside is a ligand capable of binding to a receptor of mannose 6-phosphate of a muscle cell for the preparation of a medicament for delivery of said oligonucleotide to a extralisosomal compartment of said muscle cell.