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Treatment of neurodegenerative disease through intracranial delivery of short interfering rna (sirna)

机译:通过颅内短干扰RNA(Sirna)的颅内治疗来治疗神经退行性疾病

摘要

The present invention provides devices, small interfering RNA, and methods for treating a neurodegenerative disorder comprising the steps of surgically implanting a catheter so that a discharge portion of the catheter lies adjacent to a predetermined infusion site in a brain, and discharging through the discharge portion of the catheter a predetermined dosage of at least one substance capable of inhibiting production of at least one neurodegenerative protein. The present invention also provides valuable small interfering RNA vectors, and methods for treating neurodegenerative disorders such as Alzheimer's disease, Parkinson's disease, Huntington's disease, Spinocerebellar Ataxia Type 1, Type 2, Type 3, and/or dentatorubral-pallidoluysian atrophy.
机译:本发明提供了用于治疗神经退行性疾病的装置,小的干扰RNA和方法,其包括以下步骤:通过外科手术植入导管,使得导管的排出部分位于脑中预定的输注部位附近,并通过排出部分排出预定剂量的导管中的至少一种能够抑制至少一种神经退行性蛋白质的产生的物质。本发明还提供了有价值的小的干扰RNA载体,以及用于治疗神经退行性疾病例如阿尔茨海默氏病,帕金森氏病,亨廷顿氏病,脊髓小脑共济失调1型,2型,3型和/或齿龈-睑板肌萎缩的方法。

著录项

  • 公开/公告号AU2003293035A8

    专利类型

  • 公开/公告日2004-06-18

    原文格式PDF

  • 申请/专利权人 MEDTRONIC INC.;

    申请/专利号AU20030293035

  • 发明设计人 WILLIAM F. KAEMMERER;

    申请日2003-11-26

  • 分类号A61K31/713;

  • 国家 AU

  • 入库时间 2022-08-21 23:01:45

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