| 发明名称 |
SELECTIVE GENE THERAPY EXPRESSION SYSTEM |
| 摘要 |
The present invention relates to an expression system for systemic administration comprising a sequence encoding a protein, said expression system allowing:
the expression at a therapeutically acceptable level of the protein in the target tissues including skeletal muscles and/or the peripheral nervous tissue; andthe expression at toxically acceptable level of the protein in tissues other than the target tissues, especially in the heart. |
| 申请公布号 |
US2016058890(A1) |
申请公布日期 |
2016.03.03 |
| 申请号 |
US201414782396 |
申请日期 |
2014.04.10 |
| 申请人 |
GENETHON ;CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE |
发明人 |
Buj Bello Ana Maria;Richard Isabelle |
| 分类号 |
A61K48/00;A61K38/46;C12N15/86;A61K38/48 |
主分类号 |
A61K48/00 |
| 代理机构 |
|
代理人 |
|
| 主权项 |
1. An expression system for systemic administration comprising a sequence encoding a protein having a therapeutic effect on neuromuscular disorders and whose expression is toxic in at least one tissue, said expression system allowing:
the expression at a therapeutically acceptable level of the protein in the target tissues including skeletal muscles and/or the peripheral nervous tissue; and the expression at toxically acceptable level of the protein in tissues other than the target tissues, especially in the heart. |
| 地址 |
Evry FR |
