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1. 发明申请

WO2009046015A3 COMBINATION THERAPIES FOR TREATING TYPE 1 DIABETES 审中-公开
标题翻译：用于治疗1型糖尿病的组合疗法
公开(公告)号：WO2009046015A3
公开(公告)日：2009-07-16
申请号：PCT/US2008078286
申请日：2008-09-30
申请人： UNIV FLORIDA , GENZYME CORP , ATKINSON MARK A , EISENBEIS SCOTT , ARMENTANO DONNA , SCARIA ABRAHAM , LODIE TRACY
发明人： ATKINSON MARK A , EISENBEIS SCOTT , ARMENTANO DONNA , SCARIA ABRAHAM , LODIE TRACY
IPC分类号： A61K38/19 , A61K39/395 , A61P3/10 , C07K14/555
CPC分类号： A61K39/39541 , A61K31/436 , A61K35/28 , A61K38/193 , A61K38/26 , A61K45/06 , A61K2300/00
摘要： In accordance with the subject invention, combination therapies can be used to modulate a patient's immune response in order to prevent, delay and/or reverse type 1 diabetes.
摘要翻译：根据本发明，组合疗法可用于调节患者的免疫应答，以防止，延迟和/或逆转1型糖尿病。

2. 发明申请

WO9936545A3 ADENOVIRAL VECTORS WITH MODIFIED CAPSID PROTEINS 审中-公开
标题翻译：具有改良CAPSID蛋白的腺病毒载体
公开(公告)号：WO9936545A3
公开(公告)日：1999-11-04
申请号：PCT/US9900913
申请日：1999-01-15
申请人： GENZYME CORP , ROMANCZUK HELEN , ARMENTANO DONNA , RIORDAN CATHERINE R O
发明人： ROMANCZUK HELEN , ARMENTANO DONNA , O'RIORDAN CATHERINE R
IPC分类号： C12N15/09 , A61K35/76 , A61K38/00 , A61K48/00 , C07K14/075 , C12N7/00 , C12N15/34 , C12N15/861 , C12N15/86
CPC分类号： C12N15/86 , A61K38/00 , A61K48/00 , C07K2319/00 , C12N2710/10343 , C12N2710/10345 , C12N2810/40 , C12N2810/405 , C12N2810/60 , C12N2810/6018
摘要： The present invention is directed to adenoviral vectors having modified capsid proteins which comprise heterologous ligands that improve and/or alter the infectious capability of the vector. Such ligands are capable of binding to target cells, and their inclusion into adenoviral vectors facilitates the binding and infectious properties of the vectors. In a preferred embodiment, the ligands are peptides, and the target cells are epithelial cells. The invention is also directed to novel heterologous ligands, to ligand-receptor complexes, and to compositions comprising the adenoviral vectors of the invention. Additional aspects of the invention include methods to use the adenoviral vectors of the invention to deliver transgenes to target cells.
摘要翻译：本发明涉及具有改良衣壳蛋白的腺病毒载体，其包含改善和/或改变载体感染能力的异源配体。这样的配体能够结合靶细胞，并且它们包含在腺病毒载体中有助于载体的结合和感染性质。在优选的实施方案中，配体是肽，靶细胞是上皮细胞。本发明还涉及新颖的异源配体，配体 - 受体复合物，以及包含本发明的腺病毒载体的组合物。本发明的其它方面包括使用本发明的腺病毒载体将转基因递送至靶细胞的方法。

3. 发明申请

WO9947180A9 CHIMERIC ADENOVIRAL VECTORS FOR TARGETED GENE DELIVERY 审中-公开
标题翻译：用于目标基因交付的CHIMERIC ADENOVIRAL VECTORS
公开(公告)号：WO9947180A9
公开(公告)日：2000-02-17
申请号：PCT/US9906101
申请日：1999-03-19
申请人： GENZYME CORP , SHANKARA SRINIVAS , ARMENTANO DONNA , ROBERTS BRUCE L
发明人： SHANKARA SRINIVAS , ARMENTANO DONNA , ROBERTS BRUCE L
IPC分类号： A61K48/00 , C12N15/861 , C07H21/04 , C07K14/00 , C12N15/63
CPC分类号： C12N15/86 , A61K48/00 , C12N2710/10322 , C12N2710/10343 , C12N2710/10345 , C12N2810/6018
摘要： The present invention provides chimeric adenoviral vectors that preferentially infect a target mammalian cell. Also provided are methods of targeting gene delivery to a specific cell type, treatment of cancer and methods of inducing a specific immune response in a subject. Pharmaceutical compositions are also provided.
摘要翻译：本发明提供优先感染靶哺乳动物细胞的嵌合腺病毒载体。还提供了将靶向基因递送至特定细胞类型，治疗癌症的方法和在受试者中诱导特异性免疫应答的方法。还提供药物组合物。

4. 发明申请

WO02095006A2 MUSCLE-SPECIFIC EXPRESSION VECTORS 审中-公开
标题翻译：肌肉特异性表达载体
公开(公告)号：WO02095006A2
公开(公告)日：2002-11-28
申请号：PCT/US0216748
申请日：2002-05-22
申请人： GENZYME CORP , SOUZA DAVID , ARMENTANO DONNA
发明人： SOUZA DAVID , ARMENTANO DONNA
IPC分类号： C12N15/09 , A01N63/00 , A61K35/76 , A61K38/00 , A61K48/00 , A61P3/06 , A61P3/10 , A61P7/00 , C12N1/20 , C12N1/21 , C12N5/10 , C12N15/00 , C12N15/85 , C12N
CPC分类号： C12N15/85 , C12N2830/008 , C12N2830/85
摘要： The invention is directed to novel combinations of muscle-specifi c enhancer and promoter elements useful for achieving persistent expression in the muscle or myocytes. The muscle-specific promoter elements are derived from a muscle creatine kinase promoter, a troponin I promoter, a skeletal alpha-actin promoter, or a desmin promoter. The muscle-specific enhancer elements are derived from either troponin I internal regulatory elements, muscle creatin e kinase enhancers, or desmin enhancers.
摘要翻译：本发明涉及用于在肌肉或肌细胞中实现持续表达的肌肉特异性增强子和启动子元件的新颖组合。肌肉特异性启动子元件来源于肌肉肌酸激酶启动子，肌钙蛋白I启动子，骨骼α-肌动蛋白启动子或结蛋白启动子。肌肉特异性增强子元件来自肌钙蛋白I内调节元件，肌肉肌酸激酶增强子或结蛋白增强子。

5. 发明申请

WO9846781A3 NOVEL TRANSGENE EXPRESSION SYSTEM FOR INCREASED PERSISTENCE 审中-公开
标题翻译：用于增加持续时间的新型传输表达系统
公开(公告)号：WO9846781A3
公开(公告)日：1999-01-21
申请号：PCT/US9807841
申请日：1998-04-14
申请人： GENZYME CORP , ARMENTANO DONNA , MARSHALL JOHN , YEW NELSON , CHENG SENG , GREGORY RICHARD J
发明人： ARMENTANO DONNA , MARSHALL JOHN , YEW NELSON , CHENG SENG , GREGORY RICHARD J
IPC分类号： C12N15/09 , A61K35/76 , A61K48/00 , A61P11/00 , C07K14/47 , C12N1/15 , C12N1/19 , C12N1/21 , C12N5/10 , C12N15/861 , C12N15/86
CPC分类号： C12N15/86 , A61K48/00 , C07K14/4712 , C12N2710/10343 , C12N2830/00 , C12N2830/15 , C12N2830/42 , C12N2830/60 , C12N2830/85
摘要： The invention is directed to a transgene expression system comprising a transcription unit which contains a transgene operably linked to expression control sequences, preferably the CMV promoter, and which is delivered simultaneously with all or part of the adenovirus E4 genomic region to a cell in order to facilitate persistent expression of the transgene. The components of the transgene expression system can be delivered by vectors including plasmids and/or viruses and may be complexed with cationic amphiphiles to facilitate entry into a cell. The invention is also directed to methods for the production of the transgene expression system. The invention is further directed to compositions that contain the transgene expression system and to methods for the use of such compositions to deliver transgenes encoding biologically active proteins to cells.

6. 发明申请

WO0136620A3 VECTORS AND TRANSGENIES WITH REGULATORY ELEMENTS FOR GENE DELIVERY TO THE LIVER 审中-公开
标题翻译：具有用于基因输送到肝脏的调节元素的载体和转基因
公开(公告)号：WO0136620A3
公开(公告)日：2002-02-14
申请号：PCT/US0031444
申请日：2000-11-15
申请人： GENZYME CORP
发明人： SOUZA DAVID W , ARMENTANO DONNA , WADSWORTH SAMUEL C
IPC分类号： A61K48/00 , C12N15/85 , C12N15/861 , C12N15/86
CPC分类号： C12N15/86 , A61K48/00 , A61K48/0058 , C12N15/85 , C12N2710/10343 , C12N2799/022 , C12N2830/008 , C12N2830/15 , C12N2830/85
摘要： The invention is directed to novel combinations of liver specific enhancers and promoter elements for achieving persistent transgene expression in the liver. The liver specific enhancer elements may be derived from either the human serum albumin, prothrombin, alpha -1microglobulin or aldolase genes in single copies or in multimerized form linked to elements derived from the cytomegalovirus intermediate early (CMV), alpha -1-antitrypsin or albumin promoters. In a preferred embodiment of the invention, an adenoviral vector comprising a liver specific enhancer/promoter combination operably linked to a transgene is administered to recipient cells. In other embodiments of the invention, adeno-associated viral vectors, retroviral vectors, lentiviral vectors or a plasmid comprising the liver specific enhancer/promoter combination linked to a transgene is administered to recipient cells. Also within the scope of the invention are promoter elements derived from the human prothrombin gene and the beta -fibrinogen gene.
摘要翻译：本发明涉及用于实现肝脏中持续转基因表达的肝特异性增强子和启动子元件的新型组合。肝脏特异性增强子元件可以衍生自与来自巨细胞病毒中间体早期（CMV），α-1-抗胰蛋白酶或白蛋白的元件连接的单拷贝或多聚体形式的人血清白蛋白，凝血酶原，α-1微球蛋白或醛缩酶基因促销员。在本发明的优选实施方案中，向受体细胞施用包含可操作地连接到转基因的肝特异性增强子/启动子组合的腺病毒载体。在本发明的其它实施方案中，将腺相关病毒载体，逆转录病毒载体，慢病毒载体或包含与转基因连接的肝特异性增强子/启动子组合的质粒施用于受体细胞。也在本发明范围内的是源自人凝血酶原基因和β-纤维蛋白原基因的启动子元件。

7. 发明申请

WO0202788A3 METHODS FOR HELPER-DEPENDENT ADENOVIRAL VECTOR PRODUCTION 审中-公开
标题翻译：用于帮助依赖性腺病毒载体生产的方法
公开(公告)号：WO0202788A3
公开(公告)日：2003-01-03
申请号：PCT/US0115784
申请日：2001-05-16
申请人： GENZYME CORP
发明人： ARMENTANO DONNA
IPC分类号： C12N15/861 , C12N15/86
CPC分类号： C12N15/86 , C12N2710/10343 , C12N2800/30
摘要： The present invention provides methods and materials for the production of helper-dependent adenovirus, such as PAV, at high titers. In one embodiment, the invention comprises methods for producing high titers of helper-dependent adenovirus comprising co-transfecting a cell permissive for production of adenovirus with: a) a helper-dependent adenoviral vector comprising inverted terminal repeats (ITRs) and packaging sequence derived from a first adenoviral serotype, and a transgene of interest flanked by said ITRs; and b) a chimeric, packaging-deficient helper adenovirus which contains adenoviral genes derived from the first adenoviral serotype, packaging sequence derived from a second adenoviral serotype, and ITRs derived from either the first or second adenoviral serotypes; and collecting virions produced thereby.
摘要翻译：本发明提供了用于以高滴度生产辅助性依赖性腺病毒如PAV的方法和材料。在一个实施方案中，本发明包括用于产生高滴度的辅助性依赖性腺病毒的方法，包括共转染允许产生腺病毒的细胞的细胞：a）包含反向末端重复序列（ITR）的辅助依赖性腺病毒载体和源自第一种腺病毒血清型，以及所述ITR侧翼的转基因; 和b）嵌合的包装缺陷型辅助性腺病毒，其包含衍生自第一腺病毒血清型的腺病毒基因，源自第二种腺病毒血清型的包装序列和源自第一或第二种腺病毒血清型的ITR; 并收集由此产生的病毒粒子。

8. 发明申请

WO9412649A3 GENE THERAPY FOR CYSTIC FIBROSIS 审中-公开
标题翻译：基因治疗CYSTIC FIBROSIS
公开(公告)号：WO9412649A3
公开(公告)日：1994-11-10
申请号：PCT/US9311667
申请日：1993-12-02
申请人： GENZYME CORP
发明人： GREGORY RICHARD J , ARMENTANO DONNA , COUTURE LARRY A , SMITH ALAN E
IPC分类号： C12N15/09 , A61K31/70 , A61K38/00 , A61K48/00 , A61P43/00 , C07H21/04 , C07K14/47 , C12N5/10 , C12N15/861 , C12N15/86 , C12N15/12
CPC分类号： C12N15/86 , A61K38/00 , A61K48/00 , C07K14/4712 , C12N2710/10343 , C12N2830/42
摘要： Gene Therapy vectors, which are especially useful for cystic fibrosis, and methods for using the vectors are disclosed. In preferred embodiments, the vectors are adenovirus-based. Advantages of adenovirus-based vectors for gene therapy are that they appear to be relatively safe and can be manipulated to encode the desired gene product and at the same time are inactivated in terms of their ability to replicate in a normal lytic viral life cycle. Additionally, adenovirus has a natural tropism for airway epithelia. Therefore, adenovirus-based vectors are particularly preferred for respiratory gene therapy applications such as gene therapy for cystic fibrosis. In one embodiment, the adenovirus-based gene therapy vector comprises an adenovirus 2 serotype genome in which the Ela and Elb regions of the genome, which are involved in early stages of viral replication have been deleted and replaced by genetic material of interest (e.g., DNA encoding the cystic fibrosis transmembrane regulator protein). In another embodiment, the adenovirus-based therapy vector is a pseudo-adenovirus (PAV). PAVs contain no potentially harmful viral genes, have a theoretical capacity for foreign material of nearly 36 kb, may be produced in reasonably high titers and maintain the tropism of the parent adenovirus for dividing and non-dividing human target cell types.

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