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1.发明申请GENE THERAPY FOR AMYOTROPHIC LATERAL SCLEROSIS AND OTHER SPINAL CORD DISORDERS 有权脊柱侧凸性脊柱炎及其他脊髓损伤的基因治疗公开(公告)号：US20100267812A1
公开(公告)日：2010-10-21
申请号：US12328267
申请日：2008-12-04
CPC分类号： A61K38/30 , A61K48/00 , A61K48/005 , A61K48/0075 , C12N2750/14143 , C12N2799/025
摘要： This disclosure provides methods and compositions for treating disorders or injuries that affect motor function and control in a subject. In one aspect, the invention a transgene product is delivered to a subject's spinal cord by administering a recombinant neurotrophic viral vector containing the transgene to the brain. The viral vector delivers the transgene to a region of the brain which is susceptible to infection by the virus and which expresses the encoded recombinant viral gene product. Also provided are compositions for delivery of a transgene product to a subject's spinal cord by administering a recombinant neurotrophic viral vector containing the transgene to the subject's brain.
摘要翻译： 本公开提供了用于治疗影响受试者的运动功能和控制的疾病或损伤的方法和组合物。 一方面，本发明通过将包含转基因的重组神经营养病毒载体给予脑，将转基因产物递送至受试者的脊髓。 病毒载体将转基因递送到易受病毒感染并表达编码的重组病毒基因产物的脑区域。 还提供了通过将含有转基因的重组神经营养病毒载体给予受试者的脑，将转基因产物递送至受试者的脊髓的组合物。
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公开(公告)号：US20090069261A1
公开(公告)日：2009-03-12
申请号：US11934148
申请日：2007-11-02
摘要： This disclosure provides methods and compositions for treating disorders or injuries that affect motor function and control in a subject. In one aspect, the invention provides a method to deliver a transgene to a subject's spinal cord by administering a recombinant neurotropic viral vector containing the transgene. The viral vector delivers the transgene to a region of the deep cerebellar nuclei region of the brain. Also provided are compositions and methods to deliver a transgene to a subject's spinal cord by administering a recombinant neurotropic viral vector containing the transgene to the motor cortex region of the subject's brain.
摘要翻译： 本公开提供了用于治疗影响受试者的运动功能和控制的疾病或损伤的方法和组合物。 一方面，本发明提供了通过施用含有转基因的重组神经营养病毒载体将转基因递送至受试者的脊髓的方法。 病毒载体将转基因递送到脑的深小脑核区域的一个区域。 还提供了通过将含有转基因的重组神经营养病毒载体施用于受试者的脑的运动皮层区域来递送转基因到受试者的脊髓的组合物和方法。
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公开(公告)号：US10744210B2
公开(公告)日：2020-08-18
申请号：US11934148
申请日：2007-11-02
IPC分类号： A61K48/00 , C12N15/864 , C07K14/475
摘要： This disclosure provides methods and compositions for treating disorders or injuries that affect motor function and control in a subject. In one aspect, the invention provides a method to deliver a transgene to a subject's spinal cord by administering a recombinant neurotropic viral vector containing the transgene. The viral vector delivers the transgene to a region of the deep cerebellar nuclei region of the brain. Also provided are compositions and methods to deliver a transgene to a subject's spinal cord by administering a recombinant neurotropic viral vector containing the transgene to the motor cortex region of the subject's brain.
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4.发明授权Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders 有权用于肌萎缩性侧索硬化症和其他脊髓障碍的基因治疗公开(公告)号：US09034836B2
公开(公告)日：2015-05-19
申请号：US12328267
申请日：2008-12-04
CPC分类号： A61K38/30 , A61K48/00 , A61K48/005 , A61K48/0075 , C12N2750/14143 , C12N2799/025
摘要： This disclosure provides methods and compositions for treating disorders or injuries that affect motor function and control in a subject. In one aspect, the invention a transgene product is delivered to a subject's spinal cord by administering a recombinant neurotrophic viral vector containing the transgene to the brain. The viral vector delivers the transgene to a region of the brain which is susceptible to infection by the virus and which expresses the encoded recombinant viral gene product. Also provided are compositions for delivery of a transgene product to a subject's spinal cord by administering a recombinant neurotrophic viral vector containing the transgene to the subject's brain.
摘要翻译： 本公开提供了用于治疗影响受试者的运动功能和控制的疾病或损伤的方法和组合物。 一方面，本发明通过将包含转基因的重组神经营养病毒载体给予脑，将转基因产物递送至受试者的脊髓。 病毒载体将转基因递送到易受病毒感染并表达编码的重组病毒基因产物的脑区域。 还提供了通过将含有转基因的重组神经营养病毒载体给予受试者的脑，将转基因产物递送至受试者的脊髓的组合物。
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公开(公告)号：US20090123451A1
公开(公告)日：2009-05-14
申请号：US12187896
申请日：2008-08-07
CPC分类号： A61K31/00 , A61K9/0024
摘要： Neurological diseases, including lysosomal storage diseases, can be successfully treated using intraventricular delivery of the therapeutic agents to bypass the blood-brain barrier. Similarly, diagnostic agents and anesthetic agents can be delivered to the brain in this manner. The administration can be performed slowly to achieve maximum effect. Such administration permits greater penetration of distal portions of the brain.
摘要翻译： 可以使用治疗剂的脑室内递送绕过血脑屏障来成功地治疗包括溶酶体贮积病的神经疾病。 类似地，诊断剂和麻醉剂可以以这种方式递送到大脑。 可以缓慢执行给药以达到最大效果。 这样的施用允许脑的远端部分更大的穿透。
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公开(公告)号：US08926967B2
公开(公告)日：2015-01-06
申请号：US12175610
申请日：2008-07-18
IPC分类号： A61K38/46
CPC分类号： A61K38/465 , A61K9/0019 , A61K38/47 , C12Y301/04012 , C12Y301/06013 , C12Y302/0102 , C12Y302/01045 , C12Y302/01076 , C12Y304/14
摘要： Lysosomal storage diseases can be successfully treated using intraventricular delivery of the enzyme which is etiologically deficient in the disease. The administration can be performed slowly to achieve maximum effect. Surprisingly, effects are seen on both sides of the blood-brain barrier, making this an ideal delivery means for lysosomal storage diseases which affect both brain and visceral organs.
摘要翻译： 可以使用致病性疾病缺陷的酶的心室内递送来成功地治疗溶酶体贮积病。 可以缓慢执行给药以达到最大效果。 令人惊讶的是，在血脑屏障的两侧看到效果，使其成为影响脑和内脏器官的溶酶体储存疾病的理想递送手段。
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公开(公告)号：US20090130079A1
公开(公告)日：2009-05-21
申请号：US12175610
申请日：2008-07-18
申请人： James Dodge , Marco Passini , Lamya Shihabuddin , Seng H. Cheng
发明人： James Dodge , Marco Passini , Lamya Shihabuddin , Seng H. Cheng
IPC分类号： A61K38/46
CPC分类号： A61K38/465 , A61K9/0019 , A61K38/47 , C12Y301/04012 , C12Y301/06013 , C12Y302/0102 , C12Y302/01045 , C12Y302/01076 , C12Y304/14
摘要： Lysosomal storage diseases can be successfully treated using intraventricular delivery of the enzyme which is etiologically deficient in the disease. The administration can be performed slowly to achieve maximum effect. Surprisingly, effects are seen on both sides of the blood-brain barrier, making this an ideal delivery means for lysosomal storage diseases which affect both brain and visceral organs.
摘要翻译： 可以使用致病性疾病缺陷的酶的心室内递送来成功地治疗溶酶体贮积病。 可以缓慢执行给药以达到最大效果。 令人惊讶的是，在血脑屏障的两侧看到效果，使其成为影响脑和内脏器官的溶酶体储存疾病的理想递送手段。
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公开(公告)号：US20090117156A1
公开(公告)日：2009-05-07
申请号：US12187844
申请日：2008-08-07
IPC分类号： A61K39/235 , A61K39/12 , A61K31/7088
CPC分类号： C12N9/16 , A01K2207/00 , A01K2227/105 , A01K2267/0306 , A61K48/0083 , C12N15/8509 , C12N15/86 , C12N2750/14143 , C12N2840/007 , G01N2500/00
摘要： This disclosure pertains to methods and compositions for tolerizing a mammal's brain to exogenously administered acid sphingomyelinase polypeptide by first delivering an effective amount of a transgene encoding the polypeptide to the mammal's hepatic tissue and then administering an effective amount of the transgene to the mammal's central nervous system (CNS).
摘要翻译： 本公开涉及通过首先将有效量的编码该多肽的转基因递送至哺乳动物的肝组织，然后向哺乳动物的中枢神经系统施用有效量的转基因，来将哺乳动物的脑耐受到外源性施用的酸性鞘磷脂酶多肽的方法和组合物 （CNS）。
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公开(公告)号：US20110038851A1
公开(公告)日：2011-02-17
申请号：US12600141
申请日：2008-05-16
IPC分类号： A61K38/47 , A61K31/7088 , A61K38/48 , A61K39/395 , A61K38/17 , A61P25/28 , A61P25/16 , A61P25/14 , A61P25/00 , A61P43/00 , C12N9/24 , C12N9/48 , C07H21/00 , C07K14/47
CPC分类号： A61K38/4873 , A61K31/00 , A61K31/047 , A61K31/4353 , A61K31/436 , A61K31/7088 , A61K38/13 , A61K38/1709 , A61K38/47 , A61K38/488 , A61K45/06 , A61K48/005 , A61K2300/00 , C12Y302/01045 , C12Y304/22041 , C12Y304/23005
摘要： This invention relates generally to treating synucleinopathies in subjects that are not clinically diagnosed with a lysosomal storage disease, as well as associated methods of making medicaments and screening methods.
摘要翻译： 本发明一般涉及治疗未临床诊断患有溶酶体贮积病的受试者的突触核蛋白病，以及制备药物和筛选方法的相关方法。
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公开(公告)号：US08454954B2
公开(公告)日：2013-06-04
申请号：US12600141
申请日：2008-05-16
IPC分类号： A61K38/47
CPC分类号： A61K38/4873 , A61K31/00 , A61K31/047 , A61K31/4353 , A61K31/436 , A61K31/7088 , A61K38/13 , A61K38/1709 , A61K38/47 , A61K38/488 , A61K45/06 , A61K48/005 , A61K2300/00 , C12Y302/01045 , C12Y304/22041 , C12Y304/23005
摘要： This invention relates generally to treating synucleinopathies in subjects that are not clinically diagnosed with a lysosomal storage disease, as well as associated methods of making medicaments and screening methods.
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