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公开(公告)号：US06458529B1
公开(公告)日：2002-10-01
申请号：US08459994
申请日：1995-06-02
申请人： Axel Kahn , Gildas Le Gal la Salle , Jacques Mallet , Michel Perricaudet , Marc Peschanski , Jean-Jacques Robert
发明人： Axel Kahn , Gildas Le Gal la Salle , Jacques Mallet , Michel Perricaudet , Marc Peschanski , Jean-Jacques Robert
IPC分类号： C12Q168
CPC分类号： C12N15/86 , A61K38/185 , A61K38/44 , A61K38/47 , A61K48/00 , C12N9/0006 , C12N2710/10343 , C12N2830/008 , C12Y114/16002 , C12Y302/01051
摘要： The invention concerns a recombinant DNA vector characterized in that it is capable of directing the expression an/or transcription of a selected nucleotide sequence in the cells of the central nervous system and in that it comprises (i) at least part of the genome of an adenovirus, including the regions required for that adenovirus to penetrate into the cells normally infectable by that adenovirus and (ii) being inserted into said part of genome of an adenovirus under the control of a promoter, either present or also inserted into said genome part and operative in said cells. This recombinant vector finds particular use in the treatment of diseases of the central nervous system, also in gene therapy.
摘要翻译： 本发明涉及一种重组DNA载体，其特征在于其能够指导所选择的核苷酸序列在中枢神经系统的细胞中的表达/转录，并且其包含（i）至少部分基因组 腺病毒，其包括该腺病毒所需的区域穿透通常可被该腺病毒感染的细胞，和（ii）插入在启动子控制下的，存在于或也插入到所述基因组部分中的腺病毒的基因组的所述部分基因组中， 在所述细胞中起作用。 该重组载体特别用于治疗中枢神经系统疾病，也用于基因治疗。
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2.发明授权Adenovirus vectors for the transfer of foreign genes into cells of the central nervous system particularly in brain 失效用于将外来基因转移到中枢神经系统细胞中的腺病毒载体，特别是在脑中公开(公告)号：US06756523B1
公开(公告)日：2004-06-29
申请号：US08460478
申请日：1995-06-02
申请人： Axel Kahn , Gildas Le Gal La Salle , Jacques Mallet , Michel Perricaudet , Marc Peschanski , Jean-Jacques Robert
发明人： Axel Kahn , Gildas Le Gal La Salle , Jacques Mallet , Michel Perricaudet , Marc Peschanski , Jean-Jacques Robert
IPC分类号： A01K6700
CPC分类号： C12N15/86 , A61K38/185 , A61K38/44 , A61K38/47 , A61K48/00 , C12N9/0006 , C12N2710/10343 , C12N2830/008 , C12Y114/16002 , C12Y302/01051
摘要： The invention concerns a recombinant DNA vector characterized in that it is capable of directing the expression an/or transcription of a selected nucleotide sequence in the cells of the central nervous system and in that it comprises (i) at least part of the genome of an adenovirus, including the regions required for that adenovirus to penetrate into the cells normally infectable by that adenovirus and (ii) being inserted into said part of genome of an adenovirus under the control of a promoter, either present or also inserted into said genome part and operative in said cells. This recombinant vector finds particular use in the treatment of diseases of the central nervous system, also in gene therapy.
摘要翻译： 本发明涉及一种重组DNA载体，其特征在于其能够指导所选择的核苷酸序列在中枢神经系统的细胞中的表达/转录，并且其包含（i）至少部分基因组 腺病毒，其包括该腺病毒所需的区域穿透通常可被该腺病毒感染的细胞，和（ii）插入在启动子控制下的，存在于或也插入到所述基因组部分中的腺病毒的基因组的所述部分基因组中， 在所述细胞中起作用。 该重组载体特别用于治疗中枢神经系统疾病，也用于基因治疗。
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3.发明授权Recombinant adenoviruses coding for basic fibroblast growth factors (BFGF) 失效编码碱性成纤维细胞生长因子（BFGF）的重组腺病毒公开(公告)号：US06685934B1
公开(公告)日：2004-02-03
申请号：US08718482
申请日：1996-10-09
申请人： Jacques Mallet , Michel Perricaudet , Emmanuelle Vigne , Frédéric Revah , Marc Abitbol , Paul Roustan
发明人： Jacques Mallet , Michel Perricaudet , Emmanuelle Vigne , Frédéric Revah , Marc Abitbol , Paul Roustan
IPC分类号： A01N6300
CPC分类号： C12N15/86 , A61K38/1825 , A61K48/00 , C07K14/503 , C12N2710/10343
摘要： Recombinant adenoviruses comprising a heterologous DNA sequence coding for basic blast growth factors (bFGF), preparation and uses thereof for the treatment and/or prevention of neurodegenerative diseases.
摘要翻译： 重组腺病毒，其包含编码基础胚芽生长因子（bFGF）的异源DNA序列，其用于治疗和/或预防神经变性疾病的制备及其用途。
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4.发明授权Recombinant adenoviruses coding for glial-derived neurotrophic factor (GDNF) 失效编码胶质神经营养因子（GDNF）的重组腺病毒公开(公告)号：US06245330B1
公开(公告)日：2001-06-12
申请号：US08716326
申请日：1996-10-04
IPC分类号： A61K4800
CPC分类号： A61K48/00 , A61K38/00 , C07K14/475 , C12N15/86 , C12N2710/10343
摘要： Recombinant adenoviruses comprising a heterologous DNA sequence coding for glial-derived neurotrophic growth factor (GDNF) are provided. The recombinant adenoviruses are useful in a method of expressing GDNF in a cell, wherein the cell is present in a mammal suffering from Parkinson's disease, comprising infecting said cell with a replication-defective recombinant adenovirus comprising a DNA sequence encoding GDNF operably linked to a promoter by administering the adenovirus into cells of the central nervous system. The recombinant adenoviruses of the invention are also useful in a method of treating Parkinson's disease comprising administering into cells of the central nervous system of a mammal suffering therefrom a replication defective recombinant adenovirus comprising ITRs, an encapsidation sequence and a DNA sequence encoding GDNF operably linked to a promoter, wherein the adenovirus E1 gene is non-functional and GDNF is expressed at a level that provides a therapeutic effect.
摘要翻译： 提供了包含编码神经胶质来源的神经营养生长因子（GDNF）的异源DNA序列的重组腺病毒。 所述重组腺病毒可用于在细胞中表达GDNF的方法，其中所述细胞存在于患有帕金森病的哺乳动物中，所述方法包括用复制缺陷型重组腺病毒感染所述细胞，所述复制缺陷型重组腺病毒包含与启动子可操作地连接的编码GDNF的DNA序列 通过将腺病毒施用于中枢神经系统的细胞。 本发明的重组腺病毒也可用于治疗帕金森病的方法，包括向患有其的哺乳动物的中枢神经系统的细胞施用包含ITR的复制缺陷型重组腺病毒，包封序列和编码GDNF的DNA序列，其可操作地连接到 启动子，其中腺病毒E1基因是非功能性的，并且GDNF以提供治疗效果的水平表达。
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公开(公告)号：US20050244381A1
公开(公告)日：2005-11-03
申请号：US11153317
申请日：2005-06-16
IPC分类号： A01K67/027 , A61K9/00 , A61K31/70 , A61K38/00 , A61K38/17 , A61K38/44 , A61K48/00 , A61L27/38 , A61P25/00 , A61P25/04 , C12N7/01 , C12N9/02 , C12N15/09 , C12N15/861 , C12N7/00
CPC分类号： A61L27/3804 , A61K38/446 , A61K48/00 , C12N9/0089 , C12N15/86 , C12N2710/10343
摘要： A defective recombinant adenovirus including at least one DNA sequence coding for all or an active part of a superoxide dismutase or a derivative thereof. The therapeutical use thereof and corresponding pharmaceutical compositions are also disclosed.
摘要翻译： 一种有缺陷的重组腺病毒，其包括至少一个编码超氧化物歧化酶的全部或活性部分的DNA序列或其衍生物。 还公开了其治疗用途和相应的药物组合物。
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6.发明申请Recombinant adenoviruses encoding glial cell neurotrophic factor (GDNF) 审中-公开编码胶质细胞神经营养因子（GDNF）的重组腺病毒公开(公告)号：US20050069523A1
公开(公告)日：2005-03-31
申请号：US10614794
申请日：2003-07-09
IPC分类号： A61K38/00 , A61K48/00 , C07K14/475 , C12N15/861 , C12N7/00
CPC分类号： C12N15/86 , A61K38/00 , A61K48/00 , C07K14/475 , C12N2710/10343
摘要： Recombinant adenoviruses comprising a heterologous DNA sequence coding for glial-derived neurotrophic factor (GDNF), preparation thereof, and use thereof for treating and/or preventing degenerative neurological diseases.
摘要翻译： 重组腺病毒，其包含编码神经胶质来源的神经营养因子（GDNF）的异源DNA序列，其制备及其用于治疗和/或预防退行性神经疾病的用途。
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公开(公告)号：US06723315B1
公开(公告)日：2004-04-20
申请号：US09254617
申请日：1999-03-22
申请人： Jacques Mallet , Philippe Kennel , Frédéric Revah , Axel Kahn , Georg Haase
发明人： Jacques Mallet , Philippe Kennel , Frédéric Revah , Axel Kahn , Georg Haase
IPC分类号： A01N6300
CPC分类号： A61K38/185 , A61K48/00
摘要： The invention concerns a novel method for treating motor neuron diseases and particularly amyotrophic lateral sclerosis. It consists more particularly in the systemic administration of expression systems of neurotrophic factors.
摘要翻译： 本发明涉及治疗运动神经元疾病，特别是肌萎缩性侧索硬化症的新方法。 它更特别地涉及神经营养因子的表达系统的全身给药。
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8.发明授权Pharmaceutical compositions and their use, namely for the treatment of neurodegenerative diseases 失效药物组合物及其用途，即用于治疗神经变性疾病
公开(公告)号：US6140112A
公开(公告)日：2000-10-31
申请号：US569149
申请日：1996-04-25
IPC分类号： C12N15/09 , A61K31/70 , A61K31/711 , A61K35/76 , A61K38/00 , A61K48/00 , A61P25/00 , C07K14/82 , C12N7/00 , C12N7/01 , C12N15/113 , C12N15/86 , C12Q1/68 , C07H21/04 , C12N15/63
CPC分类号： C12N15/1135 , C12N15/113 , A61K38/00 , C12N2310/111 , C12N2310/13
摘要： The present invention pertains to the use of compounds affecting the activity of transcription factors for the preparation of a pharmaceutical composition for the treatment of neurodegenerative diseases.
摘要翻译： PCT No.PCT / FR94 / 00771 Sec。 371日期：1996年4月25日 102（e）日期1996年4月25日PCT提交1994年6月27日PCT公布。 出版物WO95 / 01429 日期1995年1月12日本发明涉及影响转录因子活性的化合物在制备用于治疗神经变性疾病的药物组合物中的用途。
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公开(公告)号：US08071753B2
公开(公告)日：2011-12-06
申请号：US12481180
申请日：2009-06-09
CPC分类号： C12N15/1138 , A01K2217/058 , A61K31/7088 , C12N15/1137 , C12N2310/14
摘要： The present invention relates to nucleic acid sequences producing at least one functional miRNA, at least one functional shRNA and/or at least one functional siRNA, said miRNA, shRNA or siRNA being designed to silence the expression of a gene that encodes a cholinergic protein. The present invention further relates to compositions and kits comprising such nucleic acid sequences as well as to uses thereof.
摘要翻译： 本发明涉及产生至少一种功能性miRNA，至少一种功能性shRNA和/或至少一种功能性siRNA的核酸序列，所述miRNA，shRNA或siRNA被设计为沉默编码胆碱能蛋白的基因的表达。 本发明还涉及包含这种核酸序列的组合物和试剂盒以及其用途。
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10.发明申请Non-Integrative And Non-Replicative Lentivirus , Preparation And Uses Thereof 有权非综合性和非复制性慢病毒，其制备和用途公开(公告)号：US20080089863A1
公开(公告)日：2008-04-17
申请号：US11628534
申请日：2005-06-24
申请人： Jacques Mallet , Che Serguera , Stephanie Philippe
发明人： Jacques Mallet , Che Serguera , Stephanie Philippe
IPC分类号： A61K35/76 , A61P25/00 , C12N15/867 , C12N5/06 , C12N7/00
CPC分类号： C12N7/00 , A61K39/12 , A61K39/21 , A61K48/00 , C07K14/005 , C12N15/86 , C12N2740/15034 , C12N2740/16034 , C12N2740/16043 , C12N2740/16045 , C12N2740/16062 , C12N2740/16122 , C12N2810/6081
摘要： The invention concerns a non-integrative and non-replicative recombinant lentivirus as well as its uses, in particular for preparing a composition for transferring genes in vitro, ex vivo or in vivo. The invention is useful for transferring genes in any mammal organism, for example in liver, muscle, pancreas or central nervous system (including the ocular sphere) tissues or cells, and in particular for treating disorders or pathologies such as, for example, central nervous system, including the ocular sphere, disorders.
摘要翻译： 本发明涉及非整合和非复制重组慢病毒及其用途，特别是用于制备用于体外，离体或体内转运基因的组合物。 本发明可用于在任何哺乳动物生物体中转运基因，例如在肝脏，肌肉，胰腺或中枢神经系统（包括眼球）组织或细胞中，特别是用于治疗疾病或病理学例如中枢神经 系统，包括眼球，失调。
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