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1. 发明申请

US20070036761A1 Lentiviral apoe2 gene therapy 审中-公开
标题翻译：慢病毒apoe2基因治疗
公开(公告)号：US20070036761A1
公开(公告)日：2007-02-15
申请号：US10595151
申请日：2004-09-24
申请人： Kelly Bales , Jean-Cosme Dodart , Steven Paul , Robert Marr , Inder Verma
发明人： Kelly Bales , Jean-Cosme Dodart , Steven Paul , Robert Marr , Inder Verma
IPC分类号： A61K48/00 , C12N15/867
CPC分类号： C12N15/86 , A61K38/1709 , A61K48/00 , C12N2740/16043 , C12N2740/16045 , C12N2760/20222 , C12N2810/6081
摘要： The present invention is a method for inhibiting or reducing disease progression in subjects suffering from conditions or diseases related to the Aβ peptide, including Alzheimer's disease, Down's syndrome, cerebral amyloid angiopathy, mild cognitive impairment, and the like. The method comprises admininstering an apoE2 lentiviral vector to the subject.
摘要翻译：本发明是一种抑制或减轻患有与Aβ肽相关的病症或疾病的疾病进展的方法，包括阿尔茨海默病，唐氏综合征，脑淀粉样血管病，轻度认知障碍等。该方法包括对受试者施用apoE2慢病毒载体。

2. 发明申请

US20050003547A1 Compositions and methods for tissue specific targeting of lentivirus vectors 失效
标题翻译：慢病毒载体组织特异性靶向的组成和方法
公开(公告)号：US20050003547A1
公开(公告)日：2005-01-06
申请号：US10760123
申请日：2004-01-16
申请人： Brian Spencer , Robert Marr , Inder Verma
发明人： Brian Spencer , Robert Marr , Inder Verma
IPC分类号： A61K48/00 , C12N15/867 , C12N5/06
CPC分类号： C12N15/86 , A61K48/00 , C12N2740/16043 , C12N2740/16045 , C12N2810/50 , C12N2810/60 , C12N2810/6054 , C12N2810/855
摘要： The invention provides a lentiviral vector containing an attachment incompetent fusogenic polypeptide and a heterologous targeting polypeptide. Also provided is a lentiviral packaging construct. The construct contains a nucleic acid encoding trans-acting factors sufficient for lentiviral vector generation and an attachment incompetent fusogenic polypeptide. A lentiviral packaging system having at least two nucleic acid vectors is further provided. The lentiviral packaging system consists of a first nucleic acid vector comprising a packaging construct encoding a trans-acting factor for lentiviral vector generation, and a second nucleic acid vector encoding an attachment incompetent fusogenic polypeptide, said at least two vectors together encoding trans-acting factors sufficient for lentiviral vector generation. The invention additionally provides a lentiviral gene delivery system having at least three nucleic acid vectors. The gene delivery system consists of: a first nucleic acid vector comprising a packaging construct encoding a trans-acting factor for lentiviral vector generation; a second nucleic acid vector comprising a fusogenic construct encoding an attachment incompetent fusogenic polypeptide, and a third nucleic acid vector comprising a lentiviral vector genome encoding lentiviral cis sequences sufficient for vector genome transduction, said at least three vectors together encoding trans-acting factors sufficient for lentiviral vector generation. Finally, methods of transducing a cell and methods of targeting a gene to a cell or tissue using the lentiviral vectors and systems of the invention are also provided.
摘要翻译：本发明提供了含有附着无能融合多肽和异源靶向多肽的慢病毒载体。还提供了一种慢病毒包装结构。该构建体含有编码足够用于慢病毒载体生成的反式作用因子的核酸和附着无能力的融合多肽。还提供了具有至少两个核酸载体的慢病毒包装系统。慢病毒包装系统由包含编码慢病毒载体生成的反式作用因子的包装构建体的第一核酸载体和编码附着无能融合多肽的第二核酸载体组成，所述至少两个载体一起编码反式作用因子足以用于慢病毒载体生成。本发明另外提供了具有至少三个核酸载体的慢病毒基因递送系统。该基因递送系统包括：第一核酸载体，其包含编码慢病毒载体生成的反式作用因子的包装构建体; 包含编码附着无能融合多肽的融合构建体的第二核酸载体和包含编码足以进行载体基因组转导的慢病毒顺式序列的慢病毒载体基因组的第三核酸载体，所述至少三个载体一起编码足够的反作用因子慢病毒载体生成。最后，还提供了使用本发明的慢病毒载体和系统转导细胞的方法和将基因靶向细胞或组织的方法。

3. 发明申请

US20050100986A1 Compositions and methods for targeting a polypeptide to the central nervous system 审中-公开
标题翻译：将多肽靶向中枢神经系统的组合物和方法
公开(公告)号：US20050100986A1
公开(公告)日：2005-05-12
申请号：US10861779
申请日：2004-06-04
申请人： Inder Verma , Brian Spencer
发明人： Inder Verma , Brian Spencer
IPC分类号： A61K20060101 , A61K38/02 , A61K38/28 , A61K38/30 , A61K47/48 , C07H21/04 , C07K14/47 , C12N9/10 , C12N9/16 , C12N15/62
CPC分类号： C12N15/62 , A61K38/02 , A61K38/28 , A61K38/30 , A61K47/64 , C07K2319/74
摘要： The invention provides a chimeric CNS targeting polypeptide having a BBB-receptor binding domain and a payload polypeptide domain. The chimeric CNS targeting polypeptide can have a BBB-receptor binding domain consisting of a receptor binding domain from ApoB, ApoE, aprotinin, lipoprotein lipase, PAI-1, pseudomonas exotoxin A, transferrin, α2-macroglobulin, insulin-like growth factor, insulin, or a functional fragment thereof. Nucleic acids encoding a chimeric CNS targeting polypeptide are also provided. Further provided is a method of delivering a polypeptide to the CNS of an individual. The method consists of administering to the individual an effective amount of a chimeric CNS targeting polypeptide, said chimeric CNS targeting polypeptide comprising a BBB-receptor binding domain and a payload polypeptide domain. The method also can deliver a polypeptide to the lysosomes of CNS cells.
摘要翻译：本发明提供了具有BBB受体结合结构域和有效载荷多肽结构域的嵌合CNS靶向多肽。嵌合CNS靶向多肽可以具有由ApoB，ApoE，抑肽酶，脂蛋白脂肪酶，PAI-1，假单胞菌外毒素A，转铁蛋白，α2-巨球蛋白，胰岛素样生长因子，胰岛素的受体结合结构域组成的BBB受体结合结构域，或其功能片段。还提供了编码嵌合CNS靶向多肽的核酸。还提供了将多肽递送至个体的CNS的方法。该方法包括向个体施用有效量的嵌合CNS靶向多肽，所述嵌合CNS靶向多肽包含BBB受体结合结构域和有效载荷多肽结构域。该方法还可以将多肽递送至CNS细胞的溶酶体。

4. 发明授权

US6013516A Vector and method of use for nucleic acid delivery to non-dividing cells 失效
标题翻译：用于将核酸递送至非分裂细胞的载体和方法
公开(公告)号：US6013516A
公开(公告)日：2000-01-11
申请号：US540259
申请日：1995-10-06
申请人： Inder Verma , Didier Trono , Luigi Naldini , Philippe Gallay
发明人： Inder Verma , Didier Trono , Luigi Naldini , Philippe Gallay
IPC分类号： C12N15/09 , A61K48/00 , C07K14/145 , C07K14/16 , C12N7/00 , C12N7/04 , C12N15/00 , C12N15/867 , C12Q1/68 , C12R1/92
CPC分类号： C12N15/86 , C07K14/005 , C12N7/00 , A61K48/00 , C12N2740/13043 , C12N2740/16021 , C12N2740/16022 , C12N2740/16043 , C12N2740/16062 , C12N2760/20222
摘要： A recombinant retrovirus capable of infecting a non-dividing cell and a method of producing such a virus is provided. The recombinant retrovirus is preferably of lentivirus origin and is useful for the treatment of a variety of disorders including neurological disorders and disorders of other non-dividing cells.
摘要翻译：提供能够感染非分裂细胞的重组逆转录病毒及其生产方法。重组逆转录病毒优选为慢病毒源，并且可用于治疗各种障碍，包括神经障碍和其他非分裂细胞的病症。

5. 发明申请

US20110262347A1 METHODS AND COMPOSITIONS FOR ENHANCED DELIVERY OF COMPOUNDS 审中-公开
标题翻译：化合物增强方法和组合物
公开(公告)号：US20110262347A1
公开(公告)日：2011-10-27
申请号：US13083176
申请日：2011-04-08
申请人： Erkki Ruoslahti , Lilach Agemy , Dinorah Friedmann-Morviniski , Venkata Ramana Kotamraju , Kazuki Sugahara , Inder Verma
发明人： Erkki Ruoslahti , Lilach Agemy , Dinorah Friedmann-Morviniski , Venkata Ramana Kotamraju , Kazuki Sugahara , Inder Verma
IPC分类号： A61K51/00 , C07K7/08 , C07K14/76 , A61K47/42 , A61K9/127 , C07F13/00 , C07H21/00 , A61K38/16 , A61K31/7088 , A61K31/28 , C07D305/14 , A61K31/337 , A61K49/00 , A61P35/00 , A61P43/00 , C07K7/06 , B82Y5/00
CPC分类号： A61K38/04 , A61K47/55 , A61K47/66 , A61K47/6923 , B82Y5/00
摘要： Disclosed are compositions and methods related to multivalent compositions targeted to cells and tissues. The disclosed targeting is useful for treatment of cancer and other diseases and disorders.
摘要翻译：公开了与针对细胞和组织的多价组合物相关的组合物和方法。所公开的靶向对于治疗癌症和其它疾病和病症是有用的。

6. 发明申请

US20080292591A1 Tumor mouse models using lentiviral vectors 审中-公开
标题翻译：使用慢病毒载体的肿瘤小鼠模型
公开(公告)号：US20080292591A1
公开(公告)日：2008-11-27
申请号：US12074854
申请日：2008-03-05
申请人： Tomotoshi Marumoto , Inder Verma , Yasushi Soda , Yifeng Xia
发明人： Tomotoshi Marumoto , Inder Verma , Yasushi Soda , Yifeng Xia
IPC分类号： A61K31/7088 , A61K35/12 , A01K67/027 , A61P35/00 , G01N33/00
CPC分类号： C12N15/86 , A01K2207/05 , A01K2217/058 , A01K2227/105 , A01K2267/0331 , C07K14/82 , C12N2740/16043 , C12N2800/30 , C12N2840/002 , C12N2840/203
摘要： This invention provides model systems for study of tumors. Model cells and model animals are prepared to mimic characteristics of natural clinical tumors. The tumor models can be contacted with putative modulators to screen for therapeutics and to help understand tumorigenic processes. Model animals can include model cells, closely correlated to the characteristics of clinical tumors, in an environment closely mimicking that of the tumors in patients. Thus, the animal models are highly representative for use in clinical and pharmacological studies.
摘要翻译：本发明提供用于研究肿瘤的模型系统。模型细胞和模型动物被准备模仿天然临床肿瘤的特征。可以将肿瘤模型与推定的调节剂接触以筛选治疗，并帮助了解致瘤过程。模型动物可以包括与临床肿瘤的特征密切相关的模型细胞，其在模拟患者肿瘤的环境中。因此，动物模型在临床和药理学研究中具有高度的代表性。

7. 发明申请

US20060198833A1 Compositions and methods for targeting a polypeptide to the central nervous system 审中-公开
标题翻译：将多肽靶向中枢神经系统的组合物和方法
公开(公告)号：US20060198833A1
公开(公告)日：2006-09-07
申请号：US11401604
申请日：2006-04-10
申请人： Inder Verma , Brian Spencer
发明人： Inder Verma , Brian Spencer
IPC分类号： A61K38/47
CPC分类号： C12N15/62 , A61K38/02 , A61K38/28 , A61K38/30 , A61K47/64 , C07K2319/74
摘要： The invention provides a chimeric CNS targeting polypeptide having a BBB-receptor binding domain and a payload polypeptide domain. The chimeric CNS targeting polypeptide can have a BBB-receptor binding domain consisting of a receptor binding domain from ApoB, ApoE, aprotinin, lipoprotein lipase, PAI-1, pseudomonas exotoxin A, transferrin, α2-macroglobulin, insulin-like growth factor, insulin, or a functional fragment thereof. Nucleic acids encoding a chimeric CNS targeting polypeptide are also provided. Further provided is a method of delivering a polypeptide to the CNS of an individual. The method consists of administering to the individual an effective amount of a chimeric CNS targeting polypeptide, said chimeric CNS targeting polypeptide comprising a BBB-receptor binding domain and a payload polypeptide domain. The method also can deliver a polypeptide to the lysosomes of CNS cells.
摘要翻译：本发明提供了具有BBB受体结合结构域和有效载荷多肽结构域的嵌合CNS靶向多肽。嵌合CNS靶向多肽可以具有由ApoB，ApoE，抑肽酶，脂蛋白脂肪酶，PAI-1，假单胞菌外毒素A，转铁蛋白，α2-巨球蛋白，胰岛素样生长因子，胰岛素的受体结合结构域组成的BBB受体结合结构域，或其功能片段。还提供了编码嵌合CNS靶向多肽的核酸。还提供了将多肽递送至个体的CNS的方法。该方法包括向个体施用有效量的嵌合CNS靶向多肽，所述嵌合CNS靶向多肽包含BBB受体结合结构域和有效载荷多肽结构域。该方法还可以将多肽递送至CNS细胞的溶酶体。

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