Modified factor VIII and factor IX genes and vectors for gene therapy
申请人:Asklepios Biopharmaceutical, Inc.
公开号:EP2848253A1
公开(公告)日:2015-03-18
The present invention relates to a modified and optimized Factor VIII or Factor IX nucleic acid for inclusion in a chimeric virus vector. Use of such vector can be used for treatment of hemophilia.
本发明涉及一种经过修饰和优化的因子 VIII 或因子 IX 核酸,可用于嵌合病毒载体。这种载体可用于治疗血友病。
Modified soluble VEGF receptor-1 genes and vectors for gene therapy
申请人:Samulski Richard J.
公开号:US10385112B2
公开(公告)日:2019-08-20
The present invention relates to a modified and optimized sFlt1 nucleic acid for inclusion in a virus vector. Use of such vectors can be used for treatment of ocular disorders causing neovascularization, such as macular degeneration.
Modified Friedreich ataxia genes and vectors for gene therapy
申请人:Bamboo Therapeutics, Inc.
公开号:US10548947B2
公开(公告)日:2020-02-04
The present invention relates to a modified FXN gene providing for increased expression of the encoded protein frataxin that can be used for treatment of Friedreich ataxia.