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1.发明公开
公开(公告)号：EP2950824A1
公开(公告)日：2015-12-09
申请号：EP14702565.4
申请日：2014-01-31
申请人： INSERM - Institut National de la Santé et de la Recherche Médicale , Centre National de la Recherche Scientifique (C.N.R.S.) , Université de Strasbourg , Cornell University , Université Paris Sud-Paris XI , APHP (Assistance Publique - Hôpitaux de Paris)
CPC分类号： C12N15/86 , A61K9/0019 , A61K31/7088 , A61K38/44 , A61K48/00 , A61K48/0016 , A61K48/005 , C07K14/47 , C12N7/00 , C12N2750/14032 , C12N2750/14071 , C12N2750/14132 , C12N2750/14143 , C12N2750/14171 , C12Y116/03001
摘要： A method for preventing or treating cardiomyopathy due to energy failure in a subject in need thereof is provided. The method comprises administering to the subject a therapeutically effective amount of a vector which comprises a nucleic acid sequence encoding a gene that can reverse energy failure. An exemplary cardiomyopathy is that which is associated with Friedreich ataxia and an exemplary nucleic acid sequence comprises a nucleic acid that encodes frataxin (FXN).
摘要翻译： 提供了一种用于在需要其的受试者的预防或治疗由于能量衰竭心肌病的方法。 该方法包括施用给所述受试者治疗有效量的包含编码基因也可逆转能量衰竭的核酸序列的载体的。 示例性心肌病与做所有这一切是弗里德赖希共济失调和示例性的核酸序列相关的包括核酸确实编码frataxin（FXN）。
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2.发明公开
公开(公告)号：EP4324845A2
公开(公告)日：2024-02-21
申请号：EP23201456.3
申请日：2014-01-31
申请人： Institut National de la Santé et de la Recherche Médicale (INSERM) , Centre National de la Recherche Scientifique (CNRS) , Université de Strasbourg , Cornell University , Université Paris-Saclay , APHP (Assistance Publique - Hôpitaux de Paris)
IPC分类号： C07K14/47
摘要： The present invention relates a method for preventing or treating cardiomyopathy due to energy failure in a subject in need thereof, comprising administering to said subject a therapeutically effective amount of a vector which comprises a nucleic acid sequence of a gene that can restore energy failure.
More particularly, the invention relates to a method for preventing or treating a cardiomyopathy associated with Friedreich ataxia in a subject in need thereof, comprising administering to said subject a therapeutically effective amount of a vector which comprises a frataxin (FXN) encoding nucleic acid.-
3.发明公开
公开(公告)号：EP4324845A3
公开(公告)日：2024-04-17
申请号：EP23201456.3
申请日：2014-01-31
申请人： Institut National de la Santé et de la Recherche Médicale (INSERM) , Centre National de la Recherche Scientifique (CNRS) , Université de Strasbourg , Cornell University , Université Paris-Saclay , APHP (Assistance Publique - Hôpitaux de Paris)
IPC分类号： A61K31/7088 , A61K38/44 , C07K14/47 , A61K48/00 , A61P9/00
CPC分类号： A61K48/005 , C07K14/47 , C12N2750/1403220130101 , C12N2750/1407120130101 , C12N2750/1414320130101 , A61K31/7088 , A61K38/44 , C12Y116/03001 , A61P9/00
摘要： The present invention relates a method for preventing or treating cardiomyopathy due to energy failure in a subject in need thereof, comprising administering to said subject a therapeutically effective amount of a vector which comprises a nucleic acid sequence of a gene that can restore energy failure.
More particularly, the invention relates to a method for preventing or treating a cardiomyopathy associated with Friedreich ataxia in a subject in need thereof, comprising administering to said subject a therapeutically effective amount of a vector which comprises a frataxin (FXN) encoding nucleic acid.-
4.发明授权EXPRESSION VECTOR FOR CHOLESTEROL 24-HYDROLASE IN THERAPY OF HUNTINGTON'S DISEASE 有权表达载体24水解酶用于治疗亨廷顿氏病
公开(公告)号：EP2627359B1
公开(公告)日：2015-12-30
申请号：EP11770760.4
申请日：2011-10-14
申请人： INSERM - Institut National de la Santé et de la Recherche Médicale , Universite d'Evry Val d'Essonne , UNIVERSITE PARIS DESCARTES , Centre National de la Recherche Scientifique (CNRS)
IPC分类号： A61K38/44 , A61K48/00 , C12N15/86 , C12N15/864 , C12N9/14
CPC分类号： A61K38/44 , A61K48/00 , C12N9/0073 , C12N9/14 , C12N15/86 , C12N15/8645 , C12N2750/14143 , C12N2840/203
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5.发明公开METHODS AND PHARMACEUTICAL COMPOSITIONS FOR EXPRESSING A POLYNUCLEOTIDE OF INTEREST IN THE PERIPHERAL NERVOUS SYSTEM OF A SUBJECT 审中-公开用于表达受试者周围神经系统中的多核苷酸的方法和药物组合物
公开(公告)号：EP3320101A1
公开(公告)日：2018-05-16
申请号：EP16736134.4
申请日：2016-07-06
申请人： INSERM (Institut National de la Santé et de la Recherche Médicale) , Universite Paris-Sud , Commissariat à l'énergie atomique et aux énergies alternatives , Assistance Publique-Hôpitaux de Paris (APHP) , Université de Montpellier
IPC分类号： C12N15/864 , A61K48/00
CPC分类号： C12N15/86 , A61K35/761 , A61K48/00 , A61K2039/525 , C12N15/113 , C12N2310/11 , C12N2310/14 , C12N2750/14143 , C12N2750/14171
摘要： The present invention relates to methods and pharmaceutical compositions for expressing a polynucleotide of interest in the peripheral nervous system of a subject. In particular, the present invention relates to a method for selectively expressing a polynucleotide of interest in the peripheral nervous system of a subject in need thereof comprising the step of transducing a peripheral nerve of the subject with an amount of an AVV9 vector containing the polynucleotide of interest.
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6.发明公开EXPRESSION VECTOR FOR CHOLESTEROL 24 -HYDROLASE IN THERAPY OF HUNTINGTON' S DISEASE 有权表面活性剂FÜR24-水解剂ZUR BEHANDLUNGDER HUNTINGTON-KRANKHEIT
公开(公告)号：EP2627359A1
公开(公告)日：2013-08-21
申请号：EP11770760.4
申请日：2011-10-14
申请人： INSERM - Institut National de la Santé et de la Recherche Médicale , Universite d'Evry Val d'Essonne , UNIVERSITE PARIS DESCARTES
IPC分类号： A61K48/00 , C12N15/86 , C12N15/864 , C12N9/14
CPC分类号： A61K38/44 , A61K48/00 , C12N9/0073 , C12N9/14 , C12N15/86 , C12N15/8645 , C12N2750/14143 , C12N2840/203
摘要： The present invention relates toavector for use in the treatment of Huntington's disease, which vector comprises a cholesterol 24-hydroxylase encoding nucleic acid.
摘要翻译： 本发明涉及用于治疗亨廷顿氏病的载体，该载体包含编码胆固醇24-羟化酶的核酸。
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7.发明授权USE OF VIRAL VECTORS CARRYING THE CYP46A1 GENE FOR THE TREATMENT OF ALZHEIMER'S DISEASE 有权携带CYP46A1基因的病毒载体在治疗阿尔茨海默病中的应用
公开(公告)号：EP2187898B8
公开(公告)日：2016-09-14
申请号：EP08804014.2
申请日：2008-09-11
IPC分类号： A61K35/76 , A61K38/44 , C12N15/864 , A61K48/00 , A61P25/28
CPC分类号： A61K38/44 , A01K2207/10 , A01K2217/05 , A01K2267/0312 , C12N15/86 , C12N2750/14132 , C12N2750/14143 , C12Y114/13098
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