发明名称 |
Mannose-6-phosphate receptor mediated gene transfer into muscle cells |
摘要 |
<p>The invention relates to glycoside-compound conjugates for use in antisense strategies, particularly in vivo antisense strategies. The conjugates comprise a glycoside linked to a compound, in which 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. <IMAGE></p> |
申请公布号 |
EP1495769(A1) |
申请公布日期 |
2005.01.12 |
申请号 |
EP20030077205 |
申请日期 |
2003.07.11 |
申请人 |
LBR MEDBIOTECH B.V. |
发明人 |
PLATENBURG, GERARD JOHANNES |
分类号 |
A61K47/48;(IPC1-7):A61K47/48;A61K31/711;A61K47/26;C12Q1/68;C07K14/72;A61K48/00 |
主分类号 |
A61K47/48 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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