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公开(公告)号：US20110293571A1
公开(公告)日：2011-12-01
申请号：US13117451
申请日：2011-05-27
CPC分类号： A61K31/711 , A61K48/005 , A61K48/0075 , A61K48/0083 , A61M5/178 , C12N15/86 , C12N2740/15043
摘要： Provided is a lentiviral vector for delivery to the brain for use in treating a neurological condition, wherein the lentiviral vector is delivered directly to the brain by delivering the lentiviral vector via six or fewer tracts per hemisphere, at a single deposit point per tract.
摘要翻译： 提供了用于递送至脑部用于治疗神经病症的慢病毒载体，其中慢病毒载体通过每个半球在每个单个沉积点通过六个或更少的束递送慢病毒载体直接递送至脑。
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公开(公告)号：US06818209B1
公开(公告)日：2004-11-16
申请号：US09701014
申请日：2000-11-22
IPC分类号： A61K4800
CPC分类号： C12N15/86 , A61K48/00 , C12N2740/13043 , C12N2740/13045 , C12N2740/15043 , C12N2740/15045 , C12N2740/16043 , C12N2740/16045 , C12N2810/60
摘要： A retroviral delivery system capable of transducing a target site is described. The retroviral delivery system comprises a first nucleotide sequence coding for at least a part of an envelope protein; and one or more other nucleotide sequences derivable from a retrovirus that ensure transduction of the target site by the retroviral delivery system; wherein the first nucleotide sequence is heterologous with respect to at least one of the other nucleotide sequences; and wherein the first nucleotide sequence codes for at least a part of a rabies G protein or a mutant, variant, derivative or fragment thereof that is capable or recognising the target site.
摘要翻译： 描述了能够转导靶位点的逆转录病毒递送系统。 逆转录病毒递送系统包含编码包膜蛋白的至少一部分的第一核苷酸序列; 以及一种或多种其它可逆转录病毒的核苷酸序列，其通过逆转录病毒递送系统确保靶位点的转导; 其中所述第一核苷酸序列相对于所述其它核苷酸序列中的至少一个是异源的; 并且其中所述第一核苷酸序列编码能够或识别所述靶位点的狂犬病G蛋白或突变体，变体，衍生物或片段的至少一部分。
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公开(公告)号：US20080269473A1
公开(公告)日：2008-10-30
申请号：US12077802
申请日：2008-03-20
申请人： Alan John Kingsman , Narry Kim , Ekaterini Kotsopoulou , Jonathan Rohll , Kyriacos A. Mitrophanous
发明人： Alan John Kingsman , Narry Kim , Ekaterini Kotsopoulou , Jonathan Rohll , Kyriacos A. Mitrophanous
IPC分类号： C07H21/00
CPC分类号： C12N7/00 , A61K48/00 , C07K14/005 , C12N15/86 , C12N2740/15022 , C12N2740/15043 , C12N2740/15052 , C12N2740/16043 , C12N2740/16122 , C12N2800/22 , C12N2830/50
摘要： A method of producing a replication defective retrovirus comprising transfecting a producer cell with the following: iii) a retroviral genome; iv) a nucleotide sequence coding for retroviral gag and pol proteins; and iii) nucleotide sequences encoding other essential viral packaging components not encoded by the nucleotide sequence of (ii); characterised in that the nucleotide sequence coding for retroviral gag and pol proteins is codon optimised for expression in the producer cell.
摘要翻译： 一种产生复制缺陷型逆转录病毒的方法，包括用以下转染生产细胞：iii）逆转录病毒基因组; iv）编码逆转录病毒gag和pol蛋白的核苷酸序列; 和iii）编码不由（ii）的核苷酸序列编码的其它基本病毒包装组分的核苷酸序列; 其特征在于编码逆转录病毒gag和pol蛋白的核苷酸序列被密码子优化用于在生产细胞中表达。
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公开(公告)号：US09738907B2
公开(公告)日：2017-08-22
申请号：US10841603
申请日：2004-05-07
申请人： Philippa Radcliffe , James E. Miskin , Fraser J. Wilkes , Kyriacos A. Mitrophanous , Susan M. Kingsman
发明人： Philippa Radcliffe , James E. Miskin , Fraser J. Wilkes , Kyriacos A. Mitrophanous , Susan M. Kingsman
IPC分类号： C12N15/867 , C12N15/11 , C12N7/00 , A61K48/00 , C12N15/86
CPC分类号： C12N15/86 , A61K48/00 , C12N2740/15043 , C12N2830/003 , C12N2830/008 , C12N2830/50 , C12N2830/85 , C12N2840/20 , C12N2840/203 , C12N2840/44
摘要： Provided is a multicistronic retroviral vector genome having a first nucleic acid sequence upstream of at least one internal regulatory element, such that the level of genomic RNA available for packaging in the absence of rev, or a functional equivalent thereof, is increased.
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公开(公告)号：US20120295960A1
公开(公告)日：2012-11-22
申请号：US13346297
申请日：2012-01-09
CPC分类号： A61K48/005 , C12N15/86 , C12N2740/15043 , C12N2740/15071 , C12N2800/22
摘要： Provided is an improved treatment for Parkinson's Disease where the efficacy of L-Dopa treatment is increased by including gene therapy in the treatment regimen. The combination therapy results in long-term improvements in response to L-Dopa and diminished side effects caused by L-Dopa.
摘要翻译： 提供了一种改进的帕金森病治疗方法，其中通过在治疗方案中包括基因治疗来增加L-多巴治疗的功效。 联合治疗导致对L-Dopa的反应的长期改善和由L-多巴引起的副作用减少。
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公开(公告)号：US20110269826A1
公开(公告)日：2011-11-03
申请号：US13128813
申请日：2009-11-11
申请人： Susan M. Kingsman , Alan J. Kingsman , Scott Ralph , Kyriacos A. Mitrophanous , Stephane Palfi , Bechir Jarraya
发明人： Susan M. Kingsman , Alan J. Kingsman , Scott Ralph , Kyriacos A. Mitrophanous , Stephane Palfi , Bechir Jarraya
CPC分类号： A61K48/005 , C12N9/0073 , C12N9/78 , C12N9/88 , C12N2799/027 , C12N2799/04 , C12N2840/206 , C12N2840/50
摘要： The present invention provides methods for: (i) treating and/or preventing Parkinson's disease in a subject without causing cognitive impairment by using dopamine replacement gene therapy to maintain or restore constant physiological dopaminergic tone in both the dorsal and ventral striatum of the subject; (ii) normalising neuronal electrical activity in basal ganglia and/or subthalamic nucleus in a Parkinson's disease subject; and (iii) treating and/or preventing dyskinesias associated with oral L-dopa administration in a Parkinson's disease subject by administration of a vector system for dopamine replacement gene therapy to the subject.
摘要翻译： 本发明提供了以下方法：（i）通过使用多巴胺替代基因治疗在受试者的背侧和腹侧纹状体中维持或恢复不断的生理多巴胺能调节，治疗和/或预防受试者中的帕金森病，而不引起认知障碍; （ii）在帕金森病受试者的基底神经节和/或丘脑底核中归一化神经元电活动; 和（iii）通过向受试者施用用于多巴胺替代基因治疗的载体系统来治疗和/或预防与帕金森病患者口服左旋多巴给药相关的运动障碍。
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公开(公告)号：US20100273996A1
公开(公告)日：2010-10-28
申请号：US12587236
申请日：2009-10-02
申请人： Alan John Kingsman , Narry Kim , Ekaterini Kotsopoulou , Jonathan Rohll , Kyriacos A. Mitrophanous
发明人： Alan John Kingsman , Narry Kim , Ekaterini Kotsopoulou , Jonathan Rohll , Kyriacos A. Mitrophanous
IPC分类号： C07H21/04
CPC分类号： C12N7/00 , A61K48/00 , C07K14/005 , C12N15/86 , C12N2740/15022 , C12N2740/15043 , C12N2740/15052 , C12N2740/16043 , C12N2740/16122 , C12N2800/22 , C12N2830/50
摘要： A method of producing a replication defective retrovirus comprising transfecting a producer cell with the following: iii) a retroviral genome; iv) a nucleotide sequence coding for retroviral gag and pot proteins; and iii) nucleotide sequences encoding other essential viral packaging components not encoded by the nucleotide sequence of (ii); characterised in that the nucleotide sequence coding for retroviral gag and pot proteins is codon optimised for expression in the producer cell.
摘要翻译： 一种产生复制缺陷型逆转录病毒的方法，包括用以下转染生产细胞：iii）逆转录病毒基因组; iv）编码逆转录病毒gag和pot蛋白的核苷酸序列; 和iii）编码不由（ii）的核苷酸序列编码的其它基本病毒包装组分的核苷酸序列; 其特征在于编码逆转录病毒gag和pot蛋白的核苷酸序列被密码子优化用于在生产细胞中表达。
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