Mannose-6-phosphate receptor mediated gene transfer into muscle cells
AssigneeProsensa B.V.
InventorGerard Platenburg
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Abstract
The invention relates to glycoside-compound conjugates for use in antisense strategies and/or gene therapy. The conjugates comprise a glycoside linked to a compound, in which the glycoside is a ligand capable of binding to a mannose-6-phosphate receptor of a muscle cell. For example the cells are muscle cells of a Duchenne Muscular Dystrophy (DMD) patient and the conjugate comprises an antisense oligonucleotide which causes exon skipping and induces or restores the synthesis of dystrophin or variants thereof.
Claims22
A method for delivering an oligonucleotide or oligonucleotide equivalent into the nucleus of cells comprising an insulin-like growth factor II/mannose-6-phosphate receptor (IGF-II/MPR), said method comprising contacting a glycoside-oligonucleotide conjugate, wherein said glycoside is a ligand capable of binding to a mannose-6-phosphate receptor with said cells.
A conjugate comprising a glycoside linked to an oligonucleotide or oligonucleotide equivalent, said glycoside being a ligand capable of binding to a mannose-6-phosphate receptor of a muscle cell and said oligonucleotide or oligonucleotide equivalent comprising at least 10 nucleotides identical or complementary to a human dystrophin gene.
Dependencies are inferred from the claim text.