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1. 发明申请

WO2011012724A1 WIDESPREAD GENE DELIVERY TO THE RETINA USING SYSTEMIC ADMINISTRATION OF AAV VECTORS 审中-公开
标题翻译：使用AAV矢量的系统管理的WIDESPREAD基因递送到RETINA
公开(公告)号：WO2011012724A1
公开(公告)日：2011-02-03
申请号：PCT/EP2010/061165
申请日：2010-07-30
申请人： ASSOCIATION INSTITUT DE MYOLOGIE , GENETHON , CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE , INSERM (Institut National de la Santé et de la Recherche Médicale) , UNIVERSITE PIERRE ET MARIE CURIE (PARIS 6) , BARKATS, Martine
发明人： BARKATS, Martine
IPC分类号： C12N15/864 , A61K48/00
CPC分类号： A61K48/0075 , C12N2750/14143
摘要： The present invention relates to compositions and methods, in particular to methods based on systemic administration of scAAV, for delivering genes to cells of the retina of mammals, and in particular to photoreceptor cells, ganglion cells, glial cells, inner nuclear layer cells or cells of the retinal pigmented epithelium.
摘要翻译：本发明涉及组合物和方法，特别涉及基于全身施用scAAV的方法，用于将基因递送至哺乳动物视网膜细胞，特别是感光细胞，神经节细胞，神经胶质细胞，内核层细胞或细胞的视网膜色素上皮。

2. 发明申请

WO2002094308A1 IMPROVED NEURONAL GENE TRANSFER 审中-公开
标题翻译：改进的神经基因转移
公开(公告)号：WO2002094308A1
公开(公告)日：2002-11-28
申请号：PCT/EP2002/005354
申请日：2002-05-15
申请人： CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE , MILLECAMPS-NAVARRO, Stéphanie , BARKATS, Martine , MALLET, Jacques
发明人： MILLECAMPS-NAVARRO, Stéphanie , BARKATS, Martine , MALLET, Jacques
IPC分类号： A61K38/18
CPC分类号： A61K48/00 , A61K38/1709 , A61K38/185
摘要： The present invention is related to compositions and methods for the delivery of nucleic acids to neurons in a mammal, and uses thereof. The present invention specifically discloses the use of compounds that cause synaptic nerve sprouting to increase neuron retrograde transport of a vector or a product (a polypeptide or a nucleic acid for example) in a mammal. The invention is also based on the use of a compound that interacts with synaptosomal associated proteins to increase neuron retrograde transport of a vector or a product such as one cited above in a mammal. The invention also relates to a product comprising a viral vector comprising a transgene and a compound that causes synaptic nerve sprouting, for sequential use for delivering said transgene to neurons by retrograde transport and its uses for the preparation of a composition used as a treatment in several neurological disorders. The methods and compositions of this invention can be used to deliver various transgenes, such as markers, vaccines, therapeutic genes etc., and are suitable for experimental, therapeutic or various other applications.
摘要翻译：本发明涉及将哺乳动物中的核酸递送给神经元的组合物和方法及其用途。本发明具体公开了使用引起突触神经发芽的化合物来增加哺乳动物中载体或产物（例如多肽或核酸）的神经元逆行转运。本发明还基于与突触体相关蛋白相互作用的化合物在哺乳动物中增加载体或产物如上文引用的产物的神经元逆行转运的用途。本发明还涉及包含病毒载体的产品，其包含转基因和引起突触神经萌芽的化合物，用于顺序用于通过逆行转运将所述转基因递送至神经元及其用于制备用于多种治疗的组合物的用途神经障碍。本发明的方法和组合物可用于递送各种转基因，例如标记物，疫苗，治疗基因等，并且适用于实验，治疗或各种其它应用。

3. 发明申请

WO2009013290A1 CNS GENE DELIVERY USING PERIPHERAL ADMINISTRATION OF AAV VECTORS 审中-公开
标题翻译： CNS基因使用AAV矢量的外部管理
公开(公告)号：WO2009013290A1
公开(公告)日：2009-01-29
申请号：PCT/EP2008/059595
申请日：2008-07-22
申请人： GENETHON , CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE , BARKATS, Martine
发明人： BARKATS, Martine
IPC分类号： C12N15/864
CPC分类号： C12N15/86 , A61K48/0075 , C12N2750/14143
摘要： The present invention relates to compositions and methods for the delivery of therapeutic proteins to the CNS using recombinant AAV vectors. More specifically, the invention relates to compositions and methods for delivering proteins into the cerebrospinal fluid of mammalian subjects through peripheral administration of AAV vectors. The invention may be used to treat various disorders of the central nervous system, including degenerative diseases and motor neuron diseases.
摘要翻译：本发明涉及使用重组AAV载体将治疗性蛋白递送至CNS的组合物和方法。更具体地，本发明涉及通过外周施用AAV载体将蛋白质递送到哺乳动物受试者的脑脊液中的组合物和方法。本发明可用于治疗中枢神经系统的各种疾病，包括退行性疾病和运动神经元疾病。

4. 发明申请

WO2009043936A1 WIDESPREAD GENE DELIVERY TO MOTOR NEURONS USING PERIPHERAL INJECTION OF AAV VECTORS 审中-公开
标题翻译：使用外周注射AAV载体的基因导入电动神经元
公开(公告)号：WO2009043936A1
公开(公告)日：2009-04-09
申请号：PCT/EP2008/063297
申请日：2008-10-03
申请人： GENETHON , CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE , BARKATS, Martine
发明人： BARKATS, Martine
IPC分类号： C12N15/864 , A61K48/00
CPC分类号： C12N15/86 , A61K48/00 , C12N2750/14143
摘要： The present invention relates to compositions and methods, in particular to methods based on systemic injection of rAAV, for delivering genes to cells of the central nervous system in mammals, such as brain neurons or glial cells, and in particular to motor neurons or glial cells of the spinal cord The invention also relates to methods of treating motor neuron disorders in mammals by expression of therapeutic genes. The invention stems from the unexpected discovery that peripheral injection of AAV vectors leads to a bypass of the blood brain barrier and a massive infection of motor neurons. The invention may be used in any mammal, including human subjects.
摘要翻译：本发明涉及组合物和方法，特别涉及基于系统性注射rAAV的方法，用于将基因递送到哺乳动物（例如脑神经元或神经胶质细胞）中的中枢神经系统的细胞，特别是运动神经元或神经胶质细胞的脊髓本发明还涉及通过治疗基因的表达来治疗哺乳动物运动神经元疾病的方法。本发明源自意想不到的发现，AAV载体的外周注射导致血脑屏障的旁路和运动神经元的大量感染。本发明可用于任何哺乳动物，包括人类受试者。

5. 发明申请

WO1996005320A1 ADENOVIRUS COMPRISING A GENE CODING FOR GLUTATHION PEROXIDASE 审中-公开
标题翻译：包含GLUTATHION过氧化物的基因编码的腺病毒
公开(公告)号：WO1996005320A1
公开(公告)日：1996-02-22
申请号：PCT/FR1995001002
申请日：1995-07-26
申请人： RHONE-POULENC RORER S.A. , BARKATS, Martine , MALLET, Jacques , REVAH, Frédéric
发明人： RHONE-POULENC RORER S.A.
IPC分类号： C12N15/86
CPC分类号： C12N15/86 , A61K38/00 , A61K48/00 , C12N9/0065 , C12N2710/10343 , C12Y111/01009
摘要： The present invention relates to a defective recombinant adenovirus comprising at least a DNA sequence coding for all or an active part of glutathion peroxidase or a derivative thereof. It also relates to their utilisation in therapy and to the corresponding pharmaceutical compositions.
摘要翻译：本发明涉及至少包含编码谷胱甘肽过氧化物酶的全部或活性部分的DNA序列或其衍生物的有缺陷的重组腺病毒。它还涉及其在治疗中的应用和相应的药物组合物。

6. 发明申请

WO1996000790A1 ADENOVIRUS INCLUDING A GENE CODING FOR A SUPEROXIDE DISMUTASE 审中-公开
标题翻译： ADENOVIRUS包括用于超氧化物歧化酶的基因编码
公开(公告)号：WO1996000790A1
公开(公告)日：1996-01-11
申请号：PCT/FR1995000854
申请日：1995-06-27
申请人： RHONE-POULENC RORER S.A. , BARKATS, Martine , MALLET, Jacques , PERRICAUDET, Michel , REVAH, Frédéric
发明人： RHONE-POULENC RORER S.A.
IPC分类号： C12N15/86
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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