首页> 外国专利> VECTORS FOR GENE DELIVERY THAT PERSIST WITHIN CELLS

VECTORS FOR GENE DELIVERY THAT PERSIST WITHIN CELLS

机译:基因递送的载体持续存在于细胞内

摘要

Disclosed herein are vectors for delivery of nucleic acid sequence into a target cell. The vectors are non-viral DNA constructs. The vectors have at least one DD-ITR, and complementary copies of the nucleic acid sequence operatively linked to regulatory elements that promote expression. The construct has covalently closed ends having a hairpin structure, and persists within the recipient cells as they divide. Delivery of the vector to the target cell results in sustained expression of the nucleic acid sequences in the target cell. Also disclosed are DNA vector constructs having at least one synthetic ITR, wherein the DNA construct forms linear DNA with hairpin covalently closed ends. Methods of generating the constructs and introducing target cells to thereby promote sustained expression of the nucleic acid sequences contained therein, are also disclosed. Further disclosed are cells and populations thereof, which contain the vectors.
机译:本文公开了用于将核酸序列递送到靶细胞中的载体。载体是非病毒DNA构建体。载体具有至少一个DD-ITR,并且与促进表达的调节元件可操作地连接的核酸序列的互补副本。构建体具有共价封闭的末端,其具有发夹结构,并且在接受者细胞中持续存在。向靶细胞输送载体导致靶细胞中核酸序列的持续表达。还公开了具有至少一种合成ITR的DNA载体构建体,其中DNA构建体形成线性DNA,具有共价封闭的末端。还公开了产生构建体和引入靶细胞的方法,从而促进其中包含的核酸序列的持续表达。进一步公开的是含有载体的细胞和群体。

著录项

  • 公开/公告号EP3810782A2

    专利类型

  • 公开/公告日2021-04-28

    原文格式PDF

  • 申请/专利权人 ASKLEPIOS BIOPHARMACEUTICAL INC.;

    申请/专利号EP20190740133

  • 发明设计人 SAMULSKI RICHARD JUDE;

    申请日2019-06-21

  • 分类号C12N15/864;C12N5/10;A61K48;

  • 国家 EP

  • 入库时间 2022-08-24 18:24:28

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