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Myogenic vector systems

机译:肌原性载体系统

摘要

This invention relates to gene therapy by using vectors which encode stable MRNA and methods of using such vectors. In particular, this invention relates to vectors which establish controlled expression of recombinant genes within tissues at certain levels. The vector includes a 5' flanking region which includes necessary sequences for expression of a nucleic acid cassette, a 3' flanking region including a 3' UTR and/or 3' NCR which stabilizes mRNA expressed from the nucleic acid cassette, and a linker which connects the 5' flanking region to a nucleic acid sequence. The linker has a position for inserting a nucleic acid cassette. The linker does not contain the coding sequence of a gene that the linker is naturally associated with. The 3' flanking region is 3' to the position for inserting the nucleic acid cassette. The expression vectors of the present invention can also be regulated by a regulatory system and/or constructed with a coating.
机译:本发明涉及通过使用编码稳定的MRNA的载体的基因治疗以及使用这种载体的方法。特别地,本发明涉及以一定水平在组织内建立重组基因受控表达的载体。该载体包括:5'侧翼区,其包括表达核酸盒的必要序列; 3'侧翼区,其包括使从核酸盒表达的mRNA稳定的3'UTR和/或3'NCR;和接头,其将5'侧翼区连接至核酸序列。接头具有用于插入核酸盒的位置。接头不包含与接头天然相关的基因的编码序列。 3'侧翼区在3'插入核酸盒的位置。本发明的表达载体还可以通过调节系统来调节和/或用涂层构建。

著录项

  • 公开/公告号JPH07500967A

    专利类型

  • 公开/公告日1995-02-02

    原文格式PDF

  • 申请/专利权人

    申请/专利号JP19930508631

  • 发明设计人

    申请日1992-11-03

  • 分类号C12N15/85;A61K31/70;A61K38/00;A61K38/04;A61K38/27;C07H21/00;

  • 国家 JP

  • 入库时间 2022-08-22 04:30:35

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