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METHODS OF TREATING KENNEDY'S DISEASE

机译:肯尼迪病的治疗方法

摘要

Certain embodiments are directed to methods of ameliorating, treating, or preventing Kennedy's Disease in a subject carrying a mutation in the Androgen Receptor (AR) gene, such as expansion of a CAG trinucleotide repeat, which is associated with Kennedy's Disease, by administering an antisense compound targeted to AR. Several embodiments provided herein relate to the discovery that antisense compounds targeting Androgen Receptor can ameliorate, treat, or prevent Kennedy's Disease in a subject carrying a mutation in the Androgen Receptor gene, such as expansion of a CAG trinucleotide repeat, which is associated with Kennedy's Disease.
机译:某些实施方案涉及通过给予反义来改善,治疗或预防携带雄激素受体(AR)基因突变(例如与肯尼迪氏病有关的CAG三核苷酸重复序列的扩增)的受试者的肯尼迪氏病的方法。针对AR的化合物。本文提供的几个实施方案涉及以下发现:靶向雄激素受体的反义化合物可以改善,治疗或预防携带雄激素受体基因突变(例如,与肯尼迪病相关的CAG三核苷酸重复序列的扩增)的肯尼迪病。 。

著录项

  • 公开/公告号EP2906225A1

    专利类型

  • 公开/公告日2015-08-19

    原文格式PDF

  • 申请/专利权人 ISIS PHARMACEUTICALS INC.;

    申请/专利号EP20130846202

  • 发明设计人 HUNG GENE;

    申请日2013-10-11

  • 分类号A61K31/7088;

  • 国家 EP

  • 入库时间 2022-08-21 15:02:10

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