EFFICIENT SYSTEMIC TREATMENT OF DYSTROPHIC PATHOLOGIES

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专利权人:

Genethon

发明人:

Dickson, George,Voit, Thomas,Moullier, Philippe,Le Guiner, Caroline

申请号：

AU2017200015

公开号：

AU2017200015A1

申请日:

2017.01.03

申请国别(地区):

AU

年份:

2017

代理人:

摘要：

#$%^&*AU2017200015A120170202.pdf#####ABSTRACT EFFICIENT SYSTEMIC TREATMENT OF DYSTROPHIC PATHOLOGIES 5 A composition comprising a gene therapy product for use in the treatment of a dystrophic disease in a subject, advantageously in humans, wherein: - the gene therapy product comprises a nucleic acid sequence encoding a functional microdystrophin - the composition is systemically administered. 10

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