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1.发明授权公开(公告)号：US11499154B2
公开(公告)日：2022-11-15
申请号：US16604121
申请日：2018-04-10
申请人： GENETHON , UNIVERSITE D'EVRY-VAL-D'ESSONNE , INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE)
发明人： Ana Maria Buj Bello
IPC分类号： C12N15/113 , A61P25/02 , A61K31/712 , A61K38/46
摘要： The present invention concerns the use of antisense oligonucleotides (AON) capable of inhibiting expression of dynamin 2, advantageously human dynamin 2, for use in the treatment of Charcot-Marie-Tooth disease (CMT) and centronuclear myopathies (CNM).
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公开(公告)号：US11104897B2
公开(公告)日：2021-08-31
申请号：US15569203
申请日：2016-04-27
申请人： GENETHON , INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) , UNIVERSITE D'EVRY VAL D'ESSONNE
IPC分类号： C12N15/113 , A61K48/00 , C12N15/11 , C12N5/10 , C12N15/10
摘要： The present invention relates to compositions and methods for the treatment of nucleotide repeat expansion disorders such as myotonic dystrophy.
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公开(公告)号：US11427824B2
公开(公告)日：2022-08-30
申请号：US16345743
申请日：2017-10-27
IPC分类号： C12N15/113 , A61P21/00 , C12N9/22 , C12N15/10 , C12N15/90
摘要： The present invention relates to compositions and methods for the treatment of myotonic dystrophy.
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4.发明申请公开(公告)号：US20170112905A1
公开(公告)日：2017-04-27
申请号：US15384976
申请日：2016-12-20
CPC分类号： A61K38/465 , A61K48/005 , A61K48/0058 , C12N7/00 , C12N9/16 , C12N2750/14143 , C12Y301/03 , C12Y301/03064
摘要： The present invention provides compositions and methods for treating a myopathy. In certain embodiments, the invention provides compositions and methods for treating, improving muscle function, and prolonging survival in a subject with X-linked myotubular myopathy (XLMTM). The present invention provides a method comprising systemic administration of a composition that induces the increased expression of myotubularin in the muscle of a subject. The invention provides sustained regional and global increases in muscle function.
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5.发明申请公开(公告)号：US20170095540A1
公开(公告)日：2017-04-06
申请号：US15220139
申请日：2016-07-26
CPC分类号： A61K38/465 , A61K48/005 , A61K48/0058 , C12N7/00 , C12N9/16 , C12N2750/14143 , C12Y301/03 , C12Y301/03064
摘要： The present invention provides compositions and methods for treating a myopathy. In certain embodiments, the invention provides compositions and methods for treating, improving muscle function, and prolonging survival in a subject with X-linked myotubular myopathy (XLMTM). The present invention provides a method comprising systemic administration of a composition that induces the increased expression of myotubularin in the muscle of a subject. The invention provides sustained regional and global increases in muscle function.
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6.发明申请公开(公告)号：US20180369343A1
公开(公告)日：2018-12-27
申请号：US15856653
申请日：2017-12-28
CPC分类号： A61K38/465 , A61K48/005 , A61K48/0058 , C12N7/00 , C12N9/16 , C12N2750/14143 , C12Y301/03 , C12Y301/03064
摘要： The present invention provides compositions and methods for treating a myopathy. In certain embodiments, the invention provides compositions and methods for treating, improving muscle function, and prolonging survival in a subject with X-linked myotubular myopathy (XLMTM). The present invention provides a method comprising systemic administration of a composition that induces the increased expression of myotubularin in the muscle of a subject. The invention provides sustained regional and global increases in muscle function.
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公开(公告)号：US09895426B2
公开(公告)日：2018-02-20
申请号：US15384976
申请日：2016-12-20
IPC分类号： A61K48/00 , A61K38/46 , C12N7/00 , C07H21/00 , C07H21/04 , C12N15/00 , C12N15/85 , C12P21/06
CPC分类号： A61K38/465 , A61K48/005 , A61K48/0058 , C12N7/00 , C12N9/16 , C12N2750/14143 , C12Y301/03 , C12Y301/03064
摘要： The present invention provides compositions and methods for treating a myopathy. In certain embodiments, the invention provides compositions and methods for treating, improving muscle function, and prolonging survival in a subject with X-linked myotubular myopathy (XLMTM). The present invention provides a method comprising systemic administration of a composition that induces the increased expression of myotubularin in the muscle of a subject. The invention provides sustained regional and global increases in muscle function.
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公开(公告)号：US20160058890A1
公开(公告)日：2016-03-03
申请号：US14782396
申请日：2014-04-10
CPC分类号： A61K48/0066 , A61K38/465 , A61K38/4873 , A61K48/0058 , A61K48/0075 , C07K14/4707 , C12N9/16 , C12N9/6472 , C12N15/86 , C12N2750/14132 , C12N2750/14143 , C12N2830/008 , C12Y301/03048 , C12Y304/22054
摘要： The present invention relates to an expression system for systemic administration comprising a sequence encoding a protein, said expression system allowing: the expression at a therapeutically acceptable level of the protein in the target tissues including skeletal muscles and/or the peripheral nervous tissue; and the expression at toxically acceptable level of the protein in tissues other than the target tissues, especially in the heart.
摘要翻译： 本发明涉及全身给药的表达系统，其包含编码蛋白质的序列，所述表达系统允许：包括骨骼肌和/或周围神经组织在内的靶组织中蛋白质的治疗可接受水平的表达; 以及在靶组织以外的组织中，特别是心脏中的蛋白质在毒性可接受水平的表达。
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公开(公告)号：US11564977B2
公开(公告)日：2023-01-31
申请号：US15856653
申请日：2017-12-28
摘要： The present invention provides compositions and methods for treating a myopathy. In certain embodiments, the invention provides compositions and methods for treating, improving muscle function, and prolonging survival in a subject with X-linked myotubular myopathy (XLMTM). The present invention provides a method comprising systemic administration of a composition that induces the increased expression of myotubularin in the muscle of a subject. The invention provides sustained regional and global increases in muscle function.
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公开(公告)号：US20180256752A1
公开(公告)日：2018-09-13
申请号：US15988728
申请日：2018-05-24
CPC分类号： A61K48/0066 , A61K38/465 , A61K38/4873 , A61K48/0058 , A61K48/0075 , C07K14/4707 , C12N9/16 , C12N9/6472 , C12N15/86 , C12N2750/14132 , C12N2750/14143 , C12N2830/008 , C12Y301/03048 , C12Y304/22054
摘要： The present invention relates to an expression system for systemic administration comprising a sequence encoding a protein, said expression system allowing: the expression at a therapeutically acceptable level of the protein in the target tissues including skeletal muscles; and the expression at toxically acceptable level of the protein in tissues other than the target tissues, especially in the heart.
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