Mannose-6-phosphate receptor mediated gene transfer into muscle cells
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United States of America Patent
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Issued Date -
Jul 6, 2006
app pub date -
Jan 5, 2005
filing date -
Jan 5, 2005
priority date (Note) -
Abandoned
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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.
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