GENETHON;CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE
发明人:
Ana Maria Buj Bello,Isabelle Richard
申请号：
US15988728
公开号：
US20180256752A1
申请日:
2018.05.24
申请国别(地区):
US
年份:
2018
代理人:
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； andthe expression at toxically acceptable level of the protein in tissues other than the target tissues, especially in the heart.
