HUNTINGTON'S DISEASE THERAPEUTIC COMPOUNDS
    22.
    发明公开
    HUNTINGTON'S DISEASE THERAPEUTIC COMPOUNDS 有权
    治疗师VERBINDUNGENFÜRHUNTINGTON-KRANKHEIT

    公开(公告)号:EP3146051A1

    公开(公告)日:2017-03-29

    申请号:EP15795373.8

    申请日:2015-05-20

    IPC分类号: C12N15/113

    摘要: The present invention is directed to RNA interference (RNAi) molecules targeted against a Huntington's disease nucleic acid sequence, and methods of using these RNAi molecules to treat Huntington's disease.

    摘要翻译: 本发明涉及针对亨廷顿氏病核酸序列的RNA干扰(RNAi)分子,以及使用这些RNAi分子治疗亨廷顿氏病的方法。

    METHODS AND COMPOSITIONS FOR TREATING BRAIN DISEASES
    24.
    发明公开
    METHODS AND COMPOSITIONS FOR TREATING BRAIN DISEASES 审中-公开
    VERFAHREN UND ZUSAMMENSETZUNGEN ZUR BEHANDLUNG VON HIRNERKRANKUNGEN

    公开(公告)号:EP3024497A2

    公开(公告)日:2016-06-01

    申请号:EP14829704.7

    申请日:2014-07-20

    IPC分类号: A61K48/00

    摘要: The present disclosure provides methods of treating a disease or delivering a therapeutic agent to a mammal comprising administering to the mammal's cisterna magna and/or ventricle an rAAV particle containing a vector comprising a nucleic acid encoding a therapeutic protein inserted between a pair of AAV inverted terminal repeats in a manner such that cells with access to the cerebrospinal fluid (CSF) express the therapeutic agent and in certain embodiments secretes the therapeutic agent into the CSF for distribution to the brain.

    摘要翻译: 本公开提供了治疗疾病或向哺乳动物递送治疗剂的方法,其包括对哺乳动物的大池和/或心室施用含有载体​​的rAAV颗粒,所述载体包含插入在一对AAV倒置末端之间的编码治疗性蛋白质的核酸 以使得具有进入脑脊液(CSF)的细胞表达治疗剂的方式重复,并且在某些实施方案中将治疗剂分泌到CSF中以分配给脑。

    ANGIOTENSINS IN MUSCULAR DYSTROPHY
    26.
    发明公开
    ANGIOTENSINS IN MUSCULAR DYSTROPHY 审中-公开
    血管紧张素在肌张力障碍中的作用

    公开(公告)号:EP2986341A1

    公开(公告)日:2016-02-24

    申请号:EP14800597.8

    申请日:2014-04-18

    IPC分类号: A61P21/00

    摘要: The present invention provides, among other things, methods of treating a muscular dystrophy including administering to a subject suffering from or susceptible to a muscular dystrophy an angiotensin (1-7) peptide. The present invention is, in part, based on the unexpected discovery that administration of an angiotensin (1-7) peptide in a muscular dystrophy animal model reduces fibrosis, restores locomotor activity and restores sympathovagal balance, which are characteristic symptoms in patients suffering from muscular dystrophy. Thus, the present invention provides a new and more effective therapy for muscular dystrophy. In some embodiments, an angiotensin (1-7) peptide includes the naturally occurring angiotensin (1-7) amino acid sequence of Asp1-Arg2-Val3-Tyr4-lle5 -His6-Pro7 (SEQID NO:1). In some embodiments, the angiotensin (1-7) peptide is a functional equivalent of SEQ ID NO:1. In some embodiments, the linear peptide has an amino acid sequence of Asp1-Arg2-Val3-Ser4-lle5-H'is6-Cys7 (SEQ ID NO:2). In some embodiments, the cyclic peptide is a 4,7-cyclized angiotensin (1-7).

    摘要翻译: 本发明提供了治疗肌营养不良症的方法,包括给患有肌营养不良症或易患肌营养不良症的受试者服用血管紧张素(1-7)肽。 本发明部分基于意外的发现,即在肌营养不良动物模型中施用血管紧张素(1-7)肽减少纤维变性,恢复运动活性并恢复交感神经平衡,这是患有肌肉的患者的特征性症状 营养不良。 因此,本发明为肌营养不良提供了新的和更有效的疗法。 在一些实施方案中,血管紧张素(1-7)肽包括天然存在的Asp1-Arg2-Val3-Tyr4-Ile5-His6-Pro7(SEQ ID NO:1)的血管紧张素(1-7)氨基酸序列。 在一些实施方案中,血管紧张素(1-7)肽是SEQ ID NO:1的功能等同物。 在一些实施方案中,线性肽具有Asp1-Arg2-Val3-Ser4-Ile5-H'is6-Cys7(SEQ ID NO:2)的氨基酸序列。 在一些实施方案中,环肽是4,7-环化的血管紧张素(1-7)。