VECTEUR POUR THÉRAPIE GÉNIQUE DE L'HÉMOPHILIE CIBLÉE SUR LE FOIE ET SES PROCÉDÉS ET SON UTILISATION

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专利权人:

Vrije Universiteit Brussel

发明人:

申请号：

EP19156735.3

公开号：

EP3513802A1

申请日:

2013.10.25

申请国别(地区):

EP

年份:

2019

代理人:

摘要：

The present invention relates to vectors containing liver-specific regulatory sequences and codon-optimized factor IX or factor VIII genes, methods employing these vectors and uses of these vectors. Expression cassettes and vectors containing these liver-specific regulatory elements and codon-optimized factor IX or factor VIII genes are also disclosed. The present invention is particularly useful for applications using gene therapy, in particular for the treatment of hemophilia A and B.

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