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Vectors for Liver-Directed Gene Therapy of Hemophilia and Methods and Use Thereof

机译：血友病肝相关基因治疗的载体及其方法和用途

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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.

机译：本发明涉及含有肝特异性调节序列和密码子最优化的因子IX或因子VIII基因的载体，采用这些载体的方法和这些载体的用途。还公开了含有这些肝特异性调节元件和密码子最优化的因子IX或因子VIII基因的表达盒和载体。本发明对于使用基因疗法的应用特别有用，特别是对于血友病A和B的治疗。

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公开/公告号US2019240350A1

专利类型
公开/公告日2019-08-08

原文格式PDF
申请/专利权人 VRIJE UNIVERSITEIT BRUSSEL;
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申请/专利号US201816229491
发明设计人 THIERRY VANDENDRIESSCHE;MARINEE CHUAH;
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申请日2018-12-21
分类号A61K48;A61K38/48;A61K38/16;C12N15/86;A61K38/36;A61K38/37;
国家 US
入库时间 2022-08-21 12:06:54

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