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; and the expression at toxically acceptable level of the protein in tissues other than the target tissues, especially in the heart. |
申请公布号 |
EP2984172(A1) |
申请公布日期 |
2016.02.17 |
申请号 |
EP20140721468 |
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 |
C12N15/864;C07K14/47;C12N9/16;C12N15/113 |
主分类号 |
C12N15/864 |
代理机构 |
|
代理人 |
|
主权项 |
|
地址 |
|