GENETHON;CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE
发明人:
Ana Maria Buj Bello,Isabelle Richard
申请号：
US14782396
公开号：
US20160058890A1
申请日:
2014.04.10
申请国别(地区):
US
年份:
2016
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 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.
