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METHODS AND COMPOSITIONS FOR THE EXPRESSION OF CONSTITUTIVELY ACTIVE RAP1A FROM A VMD2 PROMOTER

机译:来自VMD2启动子组成型活性RAP1a的方法和组合物

摘要

Disclosed are nucleic acid constructs comprising a nucleic acid sequence encoding a vitelliform macular dystrophy-2 (VMD2) promoter operably linked to a nucleic acid sequence encoding Rap1a. Disclosed are vectors comprising the nucleic acid constructs disclosed herein. Disclosed are compositions comprising the disclosed nucleic acid constructs or vectors. Also disclosed are methods of treating a subject having age-related macular degeneration comprising administering one or more of the disclosed nucleic acid constructs, vectors, or compositions to a subject in need thereof.
机译:公开了核酸构建体,其包含编码与编码RAP1a的核酸序列可操作地连接的患者型黄斑营养不良-2(VMD2)启动子的核酸序列。 公开了包含本文公开的核酸构建体的载体。 公开了包含所公开的核酸构建体或载体的组合物。 还公开了治疗具有年龄相关性黄斑变性的受试者的方法,包括将一种或多种公开的核酸构建体,载体或组合物施用给有此需要的受试者。

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