首页> 外国专利> GENE EXPRESSION BY POSITIVE FEEDBACK ACTIVATION OF A CELL TYPE-SPECIFIC PROMOTER

GENE EXPRESSION BY POSITIVE FEEDBACK ACTIVATION OF A CELL TYPE-SPECIFIC PROMOTER

机译:通过细胞类型特异性启动子的正反馈激活进行基因表达

摘要

A nucleic acid construct is described which provides cell type-specific expression of a therapeutic transgene. The construct utilizes a cell type-specific promoter which drives expression of the transgene. A positive feedback loop is introduced through the addition of an amplification promoter element operably linked to the therapeutic transgene and by providing, either as part of the same construct, or in a different construct, a transcription activator for activating the amplification promoter element. In one embodiment, the amplification promoter element is a heat shock response element (HSE) and the transcription activator is HSF-1. The construct enables functional targeting of a therapeutic gene while avoiding undesirable effects in non-targeted cells, by combining sufficiently high-level expression to promote a desirable therapeutic outcome with exceptional tissue specificity. A series of promoter elements, constructs, vectors, and therapeutic approaches is presented for gene therapy of tumors such as melanoma and other genetic diseases.
机译:描述了提供治疗性转基因的细胞类型特异性表达的核酸构建体。该构建体利用驱动转基因表达的细胞类型特异性启动子。通过添加与治疗性转基因可操作连接的扩增启动子元件并通过提供作为相同构建体的一部分或在不同构建体中的转录激活剂以激活扩增启动子元件,来引入正反馈环。在一个实施方案中,扩增启动子元件是热休克响应元件(HSE),转录激活子是HSF-1。通过结合足够高水平的表达以促进具有优异的组织特异性的期望的治疗结果,该构建体使得能够对治疗基因进行功能靶向,同时避免在非靶向细胞中产生不良影响。提出了一系列启动子元件,构建体,载体和治疗方法,用于肿瘤例如黑素瘤和其他遗传疾病的基因治疗。

著录项

  • 公开/公告号EP1478750B1

    专利类型

  • 公开/公告日2012-08-08

    原文格式PDF

  • 申请/专利权人 MAYO FOUNDATION;

    申请/专利号EP20000983755

  • 发明设计人 GOUGH MICHAEL;VILE RICHARD;

    申请日2000-11-22

  • 分类号C12N15/11;C12N15/63;C07H21/02;A61K48/00;C12N15/67;C12N15/85;

  • 国家 EP

  • 入库时间 2022-08-21 17:18:00

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