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21.发明授权TRICYCLO-DNA ANTISENSE OLIGONUCLEOTIDES, COMPOSITIONS, AND METHODS FOR THE TREATMENT OF DISEASE 有权三环DNA反义寡核苷酸，组合物和治疗疾病方法
公开(公告)号：EP2417257B1
公开(公告)日：2016-03-09
申请号：EP10713338.1
申请日：2010-04-09
申请人： Association Institut de Myologie , Université Pierre et Marie Curie , Universität Bern , Centre National de la Recherche Scientifique
IPC分类号： C12N15/113 , A61K31/712
CPC分类号： C12N15/113 , A61K31/711 , A61K31/712 , C12N15/111 , C12N2310/11 , C12N2310/3231 , C12N2320/33
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22.发明公开IN VITRO GENETIC DIAGNOSTIC OF INHERITED NEUROMUSCULAR DISORDERS 审中-公开血管内皮生长因子NEEROMUSKULÄRERSTÖRUNGEN
公开(公告)号：EP2893038A1
公开(公告)日：2015-07-15
申请号：EP13759194.7
申请日：2013-09-06
申请人： INSERM - Institut National de la Santé et de la Recherche Médicale , Université d'Aix Marseille , Centre National de la Recherche Scientifique (CNRS) , Université de Montpellier I , Université Paris Descartes - Paris V , Assistance Publique Hôpitaux de Marseille , GENETHON , University College London , Association Institut de Myologie
发明人： SOULARUE, Pascal , ATLAN, David , ALLAMAND, Valérie , BARTOLI, Marc , BEROUD, Christophe , BONNE, Gisèle , BOURGEOIS, Patrice , CIRAK, Sebahattin , COSSEE, Mireille , DE CID, Rafael , KRAHN, Martin , LEYV, Nicolas , MUNTONI, Francesco , RICHARD, Isabelle
IPC分类号： C12Q1/68
CPC分类号： C12Q1/6883 , C12Q1/6809 , C12Q1/6827 , C12Q1/6874 , C12Q2600/112 , C12Q2600/156 , C12Q2600/16 , C12Q2537/16 , C12Q2539/115
摘要： The present invention provides a method for determining all the molecular causes of Inherited Neuromuscular Disorders comprising determining a number of copy number variation(s), and/or determining a number of point mutation(s) on a physiological sample comprising a genome of a subject.
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公开(公告)号：EP2766479A1
公开(公告)日：2014-08-20
申请号：EP12770503.6
申请日：2012-10-12
申请人： Association Institut de Myologie , Universität Bern , Université Pierre et Marie Curie (Paris 6) , Institut National de la Santé et de la Recherche Médicale (INSERM) , Centre National de la Recherche Scientifique
发明人： LEUMANN, Christian , GARCIA, Luis , VOIT, Thomas
IPC分类号： C12N15/113 , C12N15/11 , A61K31/712
CPC分类号： C12N15/113 , C12N15/111 , C12N2310/11 , C12N2310/315 , C12N2310/3231 , C12N2320/33
摘要： The present invention relates to a nucleic acid molecule containing a sequence of tricyclo nucleosides joined by internucleoside phosphorothioate linkage. The invention also relates to synthetic antisense oligonucleotides and to methods employing the same.
摘要翻译： 本发明涉及含有通过核苷间磷酸酯键连接的三环核苷序列的核酸分子。 本发明还涉及合成反义寡核苷酸和涉及其的方法。
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公开(公告)号：EP2547768A1
公开(公告)日：2013-01-23
申请号：EP11710168.3
申请日：2011-03-17
发明人： GARCIA, Luis , FURLING, Denis , BELEY, Cyriaque , VOIT, Thomas
IPC分类号： C12N15/11 , A61K31/7088 , A61K48/00
CPC分类号： C12N15/111 , C12N2310/11 , C12N2310/531 , C12N2320/33 , C12N2320/34 , C12N2320/50 , C12N2330/51
摘要： The present invention relates to a method to improve the activity of engineered U7 snRNAs used in the context of RNA-based therapeutics; particularly in exon skipping, exon inclusion, and mRNA eradication strategies. The resulting modified U7 snRNAs are useful for treating neuromuscular diseases, in particular Duchenne neuromuscular dystrophy, myotonic dystrophy DM1 and spinal muscular atrophy.
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25.发明公开
公开(公告)号：EP2486136A1
公开(公告)日：2012-08-15
申请号：EP10768910.1
申请日：2010-10-08
申请人： Association Institut de Myologie , Centre National de la Recherche Scientifique CNRS , Institut National de la Santé et de la Recherche Médicale , Université Pierre et Marie Curie Paris 6
发明人： GARCIA, Luis , LORAIN, Stéphanie
CPC分类号： C07K14/4708 , A61K48/005 , C12N15/85 , C12N2840/445 , C12N2840/85
摘要： The present invention provides methods and compositions for generating novel nucleic acid molecules through targeted spliceosome mediated simple or double trans-splicing. The compositions of the invention include pre-trans-splicing molecules (PTMs) designed to interact with a target precursor messenger RNA molecule (target pre- mRNA) and to mediate a simple or double trans-splicing reaction resulting in the generation of a novel chimeric RNA molecule (chimeric RNA).
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公开(公告)号：EP4422634A2
公开(公告)日：2024-09-04
申请号：EP22812555.5
申请日：2022-10-27
申请人： Association Institut de Myologie , SORBONNE UNIVERSITE , Institut National de la Santé et de la Recherche Médicale (INSERM)
IPC分类号： A61K31/506 , A61K31/00 , A61K31/36 , A61K31/4025 , A61K31/405 , A61K31/422 , A61K31/496 , A61K31/505 , A61K45/06 , A61K48/00 , A61P21/00 , G01N33/74 , G01N33/68 , A01K67/027
CPC分类号： A61K31/506 , A61K31/4025 , A61K31/505 , A61K31/422 , A61K31/496 , A61K31/36 , A61K31/405 , A61K31/00 , A61K45/06 , G01N33/74 , A61P21/00 , G01N2333/575420130101 , G01N33/6893 , G01N2800/5220130101 , G01N2800/705220130101 , A01K67/0271 , A01K2207/1220130101 , A01K2207/1520130101
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公开(公告)号：EP3589311A1
公开(公告)日：2020-01-08
申请号：EP18708408.2
申请日：2018-03-01
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公开(公告)号：EP3253874A1
公开(公告)日：2017-12-13
申请号：EP16703768.8
申请日：2016-02-08
申请人： Association Institut de Myologie , Institut National de la Santé et de la Recherche Médicale , Université Pierre et Marie Curie (Paris 6) , Centre National de la Recherche Scientifique
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , A61K31/713 , C12N2310/13 , C12N2310/3183 , C12N2310/346 , C12N2310/532 , C12N2310/321 , C12N2310/3521
摘要： The present invention relates to nucleic acids, compositions and methods for the treatment of facioscapulohumeral dystrophy.
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30.发明授权
公开(公告)号：EP2486136B1
公开(公告)日：2016-03-30
申请号：EP10768910.1
申请日：2010-10-08
申请人： Association Institut de Myologie , Centre National de la Recherche Scientifique (CNRS) , Institut National de la Santé et de la Recherche Médicale , Université Pierre et Marie Curie (Paris 6)
发明人： GARCIA, Luis , LORAIN, Stéphanie
CPC分类号： C07K14/4708 , A61K48/005 , C12N15/85 , C12N2840/445 , C12N2840/85
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