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COMPOSITIONS AND METHODS FOR INHIBITING HMGB1 EXPRESSION

机译:用于抑制HMGB1表达的组合物和方法

摘要

This disclosure relates to oligonucleotides, compositions and methods useful for reducing HMGB1 expression, particularly in hepatocytes. Disclosed oligonucleotides for the reduction of HMGB1 expression may be either double-stranded or single-stranded and may be modified for improved characteristics such as stronger resistance to nucleases and lower immunogenicity. Disclosed oligonucleotides for the reduction of HMGB1 expression may also be designed to include targeting ligands to target a particular cell or organ, such as the hepatocytes of the liver, and may be used to treat liver fibrosis and related conditions.
机译:本公开涉及用于还原HMGB1表达的寡核苷酸,组合物和方法,特别是在肝细胞中。 所公开了用于还原HMGB1表达的寡核苷酸可以是双链或单链的,可以改性以改善诸如较强的核酸血糖和较低免疫原性的特性。 所公开了用于减少HMGB1表达的寡核苷酸,也可以设计成包括靶向配体以靶向特定的细胞或器官,例如肝细胞的肝细胞,并且可用于治疗肝纤维化和相关条件。

著录项

  • 公开/公告号EP3883581A1

    专利类型

  • 公开/公告日2021-09-29

    原文格式PDF

  • 申请/专利权人 DICERNA PHARMACEUTICALS INC.;

    申请/专利号EP20190902219

  • 发明设计人 ABRAMS MARC;CHOPDA GIRISH;PARK JIHYE;

    申请日2019-12-20

  • 分类号A61K31/7088;C07H21/04;C12N15/11;C12N15/113;

  • 国家 EP

  • 入库时间 2022-08-24 21:20:48

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