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1. 发明申请

US20130209410A1 EXPRESSION VECTOR FOR CHOLESTEROL 24 -HYDROLASE IN THERAPY OF HUNTINGTON'S DISEASE 有权
标题翻译：霍奇顿病治疗中胆固醇24 -HDR的表达载体
公开(公告)号：US20130209410A1
公开(公告)日：2013-08-15
申请号：US13878845
申请日：2011-10-14
申请人： Jocelyne Caboche , Patrick Aubourg , Nathalie Cartier , Sandrine Betuing
发明人： Jocelyne Caboche , Patrick Aubourg , Nathalie Cartier , Sandrine Betuing
IPC分类号： A61K38/44 , A61K48/00 , C12N15/864
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 to a vector 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。

2. 发明授权

US09132173B2 Expression vector for cholesterol 24-hydrolase in therapy of Huntington's disease 有权
标题翻译：胆固醇24水解酶在亨廷顿氏病治疗中的表达载体
公开(公告)号：US09132173B2
公开(公告)日：2015-09-15
申请号：US13878845
申请日：2011-10-14
申请人： Jocelyne Caboche , Patrick Aubourg , Nathalie Cartier , Sandrine Betuing
发明人： Jocelyne Caboche , Patrick Aubourg , Nathalie Cartier , Sandrine Betuing
IPC分类号： A61K48/00 , A61K38/44 , C12N9/14 , C12N15/86 , C12N15/864
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 to a vector 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。

3. 发明申请

US20050032219A1 Methods of administering vectors to synaptically connected neurons 审中-公开
标题翻译：向突触连接的神经元施用载体的方法
公开(公告)号：US20050032219A1
公开(公告)日：2005-02-10
申请号：US10482696
申请日：2002-07-03
申请人： Patrick Aubourg , Nathalie Cartier-Lacave , Elise Flavigny
发明人： Patrick Aubourg , Nathalie Cartier-Lacave , Elise Flavigny
IPC分类号： A61K38/17 , A61K48/00 , C12N15/864 , C12N15/861
CPC分类号： C12N15/86 , A61K38/1709 , A61K48/00 , A61K48/0075 , C12N2750/14143
摘要： The present invention relates generally to efficient delivery of viral vectors to cells of the CNS, particularly useful in the treatment of neurodegenerative disorders and motor neuron diseases. The invention involves selecting a first population and a second population of synaptically connected neurons, wherein a therapeutic polypeptide is to be expressed in said second population of neurons; and administering rAAV virions comprising a therapeutic gene to said first subpopulation of neurons of said subject such that the rAAV virions are transported across a synapse between synaptically connected neurons. In another aspect the present invention also comprises the use of rAAV virions carrying a transgene in the preparation of a medicament for the treatment of a disease in a subject, wherein a first population and a second population of synaptically connected neurons are selected and a therapeutic polypeptide is to be expressed in said second population of neurons; and a medicament comprising recombinant adeno-associated virus (rAAV) virions is delivered to said first population of neurons of the subject, wherein said virions comprise a nucleic acid sequence that is expressible in transduced cells to provide a therapeutic effect in the subject, and wherein said rAAV virions are capable of transducing a synaptically connected neurons.
摘要翻译：本发明一般涉及向CNS细胞有效递送病毒载体，特别可用于治疗神经变性疾病和运动神经元疾病。本发明涉及选择第一群体和第二群突触连接的神经元，其中治疗性多肽将在所述第二群神经元中表达; 以及将包含治疗基因的rAAV病毒粒子施用于所述受试者的神经元的所述第一亚群，使得rAAV病毒粒子被跨越突触连接的神经元之间的突触传输。在另一方面，本发明还包括使用携带转基因的rAAV病毒颗粒在制备用于治疗受试者中的疾病的药物中的用途，其中选择第一群体和第二群突触连接的神经元，并且治疗性多肽将在所述第二群神经元中表达; 并且将包含重组腺相关病毒（rAAV）病毒粒子的药物递送至受试者的神经元的所述第一群体，其中所述病毒粒子包含在转导的细胞中可表达以在受试者中提供治疗效果的核酸序列，其中所述rAAV病毒粒子能够转导突触连接的神经元。

4. 发明申请

US20110034540A1 Use of the CYP46A1 Gene for the Treatment of Alzheimer's Disease 有权
标题翻译：使用CYP46A1基因治疗阿尔茨海默病
公开(公告)号：US20110034540A1
公开(公告)日：2011-02-10
申请号：US12678020
申请日：2008-09-11
申请人： Patrick Aubourg , Nathalie Cartier-Lacave , Eloise Hudry
发明人： Patrick Aubourg , Nathalie Cartier-Lacave , Eloise Hudry
IPC分类号： A61K31/7088 , C12N15/63 , A61P25/28
CPC分类号： A61K38/44 , A01K2207/10 , A01K2217/05 , A01K2267/0312 , C12N15/86 , C12N2750/14132 , C12N2750/14143 , C12Y114/13098
摘要： The invention relates to a viral vector for treating Alzheimers disease, which vector comprises a cholesterol 24-hydroxylase (CYP46A1) encoding nucleic acid. In a preferred embodiment, the viral vector may be an Adeno-Associated-Virus (AAV) vector, preferably an AVV5 vector. The vector may be useful for the manufacture of a pharmaceutical composition for the treatment of Alzheimers disease in a subject, wherein the vector is to be administered directly into the brain of the subject or by intravenous or intrathecal injection.
摘要翻译：本发明涉及用于治疗阿尔茨海默病的病毒载体，该载体包含编码核酸的胆固醇24-羟化酶（CYP46A1）。在优选的实施方案中，病毒载体可以是腺相关病毒（AAV）载体，优选AVV5载体。载体可用于制备用于治疗受试者中的阿尔茨海默病的药物组合物，其中所述载体将直接施用于受试者的脑中或通过静脉内或鞘内注射施用。

5. 发明授权

US08198257B2 CYP46A1 gene for the treatment of alzheimer's disease 有权
标题翻译： CYP46A1基因治疗阿尔茨海默病
公开(公告)号：US08198257B2
公开(公告)日：2012-06-12
申请号：US12678020
申请日：2008-09-11
申请人： Patrick Aubourg , Nathalie Cartier-Lacave , Eloise Hudry
发明人： Patrick Aubourg , Nathalie Cartier-Lacave , Eloise Hudry
IPC分类号： A61K48/00
CPC分类号： A61K38/44 , A01K2207/10 , A01K2217/05 , A01K2267/0312 , C12N15/86 , C12N2750/14132 , C12N2750/14143 , C12Y114/13098
摘要： The invention relates to a viral vector for treating Alzheimers disease, which vector comprises a cholesterol 24-hydroxylase (CYP46A1) encoding nucleic acid. In a preferred embodiment, the viral vector may be an Adeno-Associated-Virus (AAV) vector, preferably an AVV5 vector. The vector may be useful for the manufacture of a pharmaceutical composition for the treatment of Alzheimers disease in a subject, wherein the vector is to be administered directly into the brain of the subject or by intravenous or intrathecal injection.
摘要翻译：本发明涉及用于治疗阿尔茨海默病的病毒载体，该载体包含编码核酸的胆固醇24-羟化酶（CYP46A1）。在优选的实施方案中，病毒载体可以是腺相关病毒（AAV）载体，优选AVV5载体。载体可用于制备用于治疗受试者中的阿尔茨海默病的药物组合物，其中所述载体将直接施用于受试者的脑中或通过静脉内或鞘内注射施用。

6. 发明申请

US20050163760A1 Use of cd34+ hematopoietic progenitor cells for the treatment of cns disorders 审中-公开
标题翻译：使用cd34 +造血祖细胞治疗cns病
公开(公告)号：US20050163760A1
公开(公告)日：2005-07-28
申请号：US10501738
申请日：2002-12-06
申请人： Nathalie Cartier-Lacave , Patrick Aubourg , Muriel Ashueur , Sonia Benhamida , Francoise Pflumio
发明人： Nathalie Cartier-Lacave , Patrick Aubourg , Muriel Ashueur , Sonia Benhamida , Francoise Pflumio
IPC分类号： A61K35/12 , A61K38/00 , A61K48/00 , A61P25/28 , C07K14/47 , C07K14/705 , C12N5/0789 , C12N5/08
CPC分类号： C07K14/47 , A61K38/00 , A61K48/00 , A61K2035/124 , C07K14/705 , C12N5/0647 , C12N2510/00 , C12N2510/02 , C12N2799/027
摘要： The present invention provides novel methods for delivering cells, particularly modified cells to the central nervous system (CNS). The purpose of this invention is to present a method that provides sustained delivery of a molecule to the central nervous system, thereby increasing the bioavailability of the molecule and lengthening the possible duration of treatment.
摘要翻译：本发明提供用于将细胞，特别是经修饰的细胞递送至中枢神经系统（CNS）的新方法。本发明的目的是提供一种提供分子到中枢神经系统的持续递送，从而增加分子的生物利用度并延长治疗持续时间的方法。

7. 发明授权

US06013769A X-linked adrenoleukodystrophy gene and corresponding protein 失效
标题翻译： X连锁肾上腺脑白质营养不良基因和相应的蛋白质
公开(公告)号：US06013769A
公开(公告)日：2000-01-11
申请号：US835734
申请日：1997-04-10
申请人： Jean-Louis Mandel , Patrick Aubourg , Jean Mosser , Claude Sarde
发明人： Jean-Louis Mandel , Patrick Aubourg , Jean Mosser , Claude Sarde
IPC分类号： A61K38/00 , C07K14/47 , C07K14/705 , C12N9/00 , G01N33/68 , C07K14/00 , A61K39/395 , C07H21/04
CPC分类号： C12N9/93 , C07K14/47 , C07K14/705 , G01N33/6893 , A61K38/00 , C12N2799/027
摘要： The present invention relates to adrenoleukodystrophy proteins and uses thereof. More particularly, the invention relates to human adrenoleukodystrophy proteins. Mutations of the adrenoleukodystrophy protein cause adrenoleukodystrophy or adrenomyelopathy.
摘要翻译：本发明涉及肾上腺脑白质营养不良蛋白及其用途。更具体地，本发明涉及人肾上腺素能营养不良蛋白。肾上腺脑白质营养不良蛋白的突变引起肾上腺脑白质营养不良或肾上腺皮质病变。

8. 发明授权

US07308803B2 Insulation system with condensate wicking for vertical applications 有权
标题翻译：绝缘系统，具有冷凝芯吸用于垂直应用
公开(公告)号：US07308803B2
公开(公告)日：2007-12-18
申请号：US10984237
申请日：2004-11-08
申请人： Matthew C. Brokaw , David D. Hokens , Patrick Aubourg
发明人： Matthew C. Brokaw , David D. Hokens , Patrick Aubourg
IPC分类号： F25D23/06 , F25D21/14
CPC分类号： F24F13/0263 , F16L59/141 , F24F6/043 , F24F13/0281 , F24F2013/225
摘要： Provided is an assembly for insulating substantially vertical runs of cooled surfaces that are continuously or periodically exposed to air or another gas that has a dewpoint temperature above that of the cooled surface, thereby resulting in condensation water vapor on or adjacent the cooled surface. One embodiment of the insulating assembly provides a wicking material layer adjacent the cooled surface that, in combination with transporting and evaporating structures, removes the condensate from the vicinity of the cooled surface and allows for its evaporation into the ambient gas surrounding the exterior surface of the insulating assembly. Another embodiment provides for exposing the condensate to an internal conditioned gas stream to provide for evaporation that does not necessarily utilize an exterior surface. Both embodiments remove condensate from the insulating structure and thereby tend to avoid any substantial condensate accumulation and maintain the insulating performance.
摘要翻译：提供了一种用于绝缘的冷却表面的大致垂直流动的组件，其连续或周期性地暴露于空气或露点温度高于冷却表面温度的另一种气体，从而导致在冷却表面上或附近冷凝水蒸气。绝缘组件的一个实施例提供了靠近冷却表面的芯吸材料层，其结合输送和蒸发结构，从冷却表面附近去除冷凝物，并允许其蒸发到环境气体周围的周围绝缘组件。另一实施例提供将冷凝物暴露于内部调节气流以提供不一定利用外表面的蒸发。两个实施方案从绝缘结构中去除冷凝物，从而倾向于避免任何明显的冷凝物积聚并保持绝缘性能。

9. 发明申请

US20060016204A1 Insulation system with condensate wicking for vertical applications 有权
标题翻译：绝缘系统，具有冷凝芯吸用于垂直应用
公开(公告)号：US20060016204A1
公开(公告)日：2006-01-26
申请号：US10984237
申请日：2004-11-08
申请人： Matthew Brokaw , David Hokens , Patrick Aubourg
发明人： Matthew Brokaw , David Hokens , Patrick Aubourg
IPC分类号： F28D15/00 , F25D23/06
CPC分类号： F24F13/0263 , F16L59/141 , F24F6/043 , F24F13/0281 , F24F2013/225
摘要： Provided is an assembly for insulating substantially vertical runs of cooled surfaces that are continuously or periodically exposed to air or another gas that has a dewpoint temperature above that of the cooled surface, thereby resulting in condensation water vapor on or adjacent the cooled surface. One embodiment of the insulating assembly provides a wicking material layer adjacent the cooled surface that, in combination with transporting and evaporating structures, removes the condensate from the vicinity of the cooled surface and allows for its evaporation into the ambient gas surrounding the exterior surface of the insulating assembly. Another embodiment provides for exposing the condensate to an internal conditioned gas stream to provide for evaporation that does not necessarily utilize an exterior surface. Both embodiments remove condensate from the insulating structure and thereby tend to avoid any substantial condensate accumulation and maintain the insulating performance.
摘要翻译：提供了一种用于绝缘的冷却表面的大致垂直流动的组件，其连续或周期性地暴露于空气或露点温度高于冷却表面温度的另一种气体，从而导致在冷却表面上或附近冷凝水蒸气。绝缘组件的一个实施例提供了邻近冷却表面的芯吸材料层，其结合输送和蒸发结构，从冷却表面附近去除冷凝物并允许其蒸发到环境气体周围的周围绝缘组件。另一实施例提供将冷凝物暴露于内部调节气流以提供不一定利用外表面的蒸发。两个实施方案从绝缘结构中去除冷凝物，从而倾向于避免任何明显的冷凝物积聚并保持绝缘性能。

10. 发明授权

US5869039A X-linked adrenoleukodystrophy gene and corresponding protein 失效
标题翻译： X连锁肾上腺脑白质营养不良基因和相应的蛋白质
公开(公告)号：US5869039A
公开(公告)日：1999-02-09
申请号：US479403
申请日：1995-06-07
申请人： Jean-Louis Mandel , Patrick Aubourg , Jean Mosser , Claude Sarde
发明人： Jean-Louis Mandel , Patrick Aubourg , Jean Mosser , Claude Sarde
IPC分类号： A61K38/00 , C07K14/47 , C07K14/705 , C12N9/00 , G01N33/68 , A01N63/00 , A61K48/00
CPC分类号： C12N9/93 , C07K14/47 , C07K14/705 , G01N33/6893 , A61K38/00 , C12N2799/027
摘要： A method for the treatment of adrenoleukodystrophy or adrenomyeloneuropathy, in which there is administered to a human patient in need of the same an effective amount of hematopoietic cells modified ex vivo by infection with a retroviral vector containing a nucleic acid fragment comprising a sequence as represented on FIG. 2 (SEQ. ID No: 1) or FIG. 6 (SEQ. ID. No: 4-17) or FIG. 7 (SEQ. ID No: 18-23). Also, human hematopoietic cells can be treated ex vivo to correct the content of very long chain fatty acids in them, by providing human hematopoietic cells of a patient affected by adrenoleukodystrophy or adrenomyeloneuropathy, and infecting them ex vivo with a retroviral vector containing, operably linked to a promoter, a nucleic acid fragment as above. The expression of the nucleic acid in the hematopoietic cells corrects the content of very long chain fatty acids in the hematopoietic cells.
摘要翻译：一种用于治疗肾上腺脑白质营养不良或肾上腺皮质神经病的方法，其中向需要相同有效量的造血细胞的人类患者施用有效量的造血细胞，所述造血细胞通过用含有如下所示序列的核酸片段的逆转录病毒载体的感染进行感染图。 2（SEQ ID NO：1）或图2。 6（SEQ ID NO：4-17）或图6。 7（SEQ ID NO：18-23）。此外，通过提供受肾上腺脑白质营养不良或肾上腺皮质神经病变影响的患者的人造血细胞，并使用含有可操作地连接的逆转录病毒载体感染它们，离体处理人类造血细胞以校正其中的非常长链脂肪酸的含量启动子，如上所述的核酸片段。造血细胞中核酸的表达校正了造血细胞中非常长链脂肪酸的含量。

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