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Treatment of down syndrome gene related diseases by inhibition of natural antisense transcript to a down syndrome gene

机译:通过抑制唐氏综合症基因的自然反义转录本来治疗唐氏综合症相关疾病

摘要

The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of a Down Syndrome Gene, in particular, by targeting natural antisense polynucleotides of a Down Syndrome Gene. The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of Down Syndrome Genes.
机译:本发明涉及特别是通过靶向唐氏综合症基因的天然反义多核苷酸来调节唐氏综合症基因的表达和/或功能的反义寡核苷酸。本发明还涉及这些反义寡核苷酸的鉴定及其在治疗与唐氏综合症基因表达有关的疾病和病症中的用途。

著录项

  • 公开/公告号US10450567B2

    专利类型

  • 公开/公告日2019-10-22

    原文格式PDF

  • 申请/专利权人 CURNA INC.;

    申请/专利号US201816026359

  • 发明设计人 JOSEPH COLLARD;OLGA KHORKOVA SHERMAN;

    申请日2018-07-03

  • 分类号C07H21/04;C12N15/113;A61K31/713;

  • 国家 US

  • 入库时间 2022-08-21 12:16:18

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