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1. 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。

2. 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。

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

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。

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。

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