首页> 外国专利> TARGETING KIT WITH SPLICE SWITCHING OLIGONUCLEOTIDES TO INDUCE APOPTOSIS OF MAST CELLS

TARGETING KIT WITH SPLICE SWITCHING OLIGONUCLEOTIDES TO INDUCE APOPTOSIS OF MAST CELLS

机译:靶向开关与寡糖交换寡糖诱导肥大细胞凋亡

摘要

Compositions and methods for modulating Kit biological activities are provided. In some embodiments, the presently disclosed subject matter provides antisense oligomers having 10 to 50 linked nucleotides, wherein the antisense oligomer is targeted to a region of a Kit-encoding pre-mRNA, and further wherein the targeted region includes sequences involved in splicing of the Kit-encoding pre-mRNA. Also provided are expression vectors encoding the antisense oligomers disclosed herein, morpholino oligomer derivatives of the presently disclosed antisense oligomers, pharmaceutical compositions that include the disclosed antisense oligomers, expression vectors, and/or morpholino oligomers, and methods for modulating splicing of a Kit pre-RNA in a cell and/or a tissue, inducing apoptosis in mast cells, and treating diseases, disorders, and/or conditions associated with Kit expression.
机译:提供了用于调节试剂盒生物活性的组合物和方法。在一些实施方案中,本发明提供了具有10至50个连接的核苷酸的反义寡聚体,其中所述反义寡聚体靶向于编码Kit的pre-mRNA的区域,并且进一步地,所述靶向区域包括与所述mRNA的剪接有关的序列。试剂盒编码pre-mRNA。还提供了编码本文公开的反义寡聚物的表达载体,目前公开的反义寡聚物的吗啉代寡聚物衍生物,包括公开的反义寡聚物的药物组合物,表达载体和/或吗啉代寡聚物,以及调节Kit pre-spice的剪接的方法。细胞和/或组织中的RNA,诱导肥大细胞凋亡,并治疗与Kit表达相关的疾病,病症和/或状况。

著录项

  • 公开/公告号WO2020046985A1

    专利类型

  • 公开/公告日2020-03-05

    原文格式PDF

  • 申请/专利权人 NORTH CAROLINA STATE UNIVERSITY;

    申请/专利号WO2019US48400

  • 发明设计人 CRUSE GLENN P.;

    申请日2019-08-27

  • 分类号C12N15/113;C07H21;C12N15/63;A61K31/7088;A61P35;A61P35/02;

  • 国家 WO

  • 入库时间 2022-08-21 11:13:01

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