首页> 外国专利> Vectors and methods and uses for liver-directed gene therapy for hemophilia

Vectors and methods and uses for liver-directed gene therapy for hemophilia

机译:血友病肝相关基因治疗的载体,方法和用途

摘要

The present invention relates to vectors containing liver-specific regulatory sequences and codon optimized Factor IX or Factor VIII genes, methods using these vectors, and uses of these vectors. Also disclosed are expression cassettes and vectors comprising these liver-specific regulatory elements and codon optimized Factor IX or Factor VIII genes. The present invention is particularly useful for applications using gene therapy, particularly 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优化的因子IX或因子VIII基因的载体,使用这些载体的方法,以及这些载体的用途。还公开了包含这些肝特异性调节元件和密码子优化的因子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。

著录项

相似文献

  • 专利
  • 外文文献
  • 中文文献
获取专利

客服邮箱:kefu@zhangqiaokeyan.com

京公网安备:11010802029741号 ICP备案号:京ICP备15016152号-6 六维联合信息科技 (北京) 有限公司©版权所有
  • 客服微信

  • 服务号