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1. 发明申请

WO2004063356A2 PERSISTENT EXPRESSION OF CANDIDATE MOLECULE IN PROLIFERATING STEM AND PROGENITOR CELLS FOR DELIVERY OF THERAPEUTIC PRODUCTS 审中-公开
标题翻译：候选化合物在促进治疗产品的干细胞和祖细胞中的表达
公开(公告)号：WO2004063356A2
公开(公告)日：2004-07-29
申请号：PCT/US2004/000929
申请日：2004-01-13
申请人： RAO, Mahendra, S. , CAPECCHI, Mario, R.
发明人： RAO, Mahendra, S. , CAPECCHI, Mario, R.
IPC分类号： C12N
CPC分类号： C12N15/907 , A61K35/12 , A61K48/00 , C12N5/0623 , C12N15/902 , C12N2510/04 , C12N2800/108 , C12N2800/30 , C12N2800/60 , C12N2830/42 , C12N2840/203 , C12N2840/44
摘要： A method of obtaining and the resulting isolated progenitor or stem cell population of proliferating cells persistently expressing a candidate molecule. Further, novel methods of ex vivo gene product ( e.g ., protein) production and treating symptoms of neurological or neurodegenerative disorders are also provided.
摘要翻译：获得和产生的增殖细胞的分离祖细胞或干细胞群的方法持续地表达候选分子。此外，还提供了离体基因产物（例如蛋白质）生产和治疗神经或神经退行性疾病症状的新方法。

2. 发明申请

WO2008098181A2 IN VIVO GENOME-WIDE MUTAGENESIS 审中-公开
标题翻译：在生殖器基因组广泛的MUTAGENESIS
公开(公告)号：WO2008098181A2
公开(公告)日：2008-08-14
申请号：PCT/US2008/053446
申请日：2008-02-08
申请人： UNIVERSITY OF UTAH RESEARCH FOUNDATION , WU, Sen , CAPECCHI, Mario, R.
发明人： WU, Sen , CAPECCHI, Mario, R.
IPC分类号： C12N15/74
CPC分类号： C12N15/1051 , C12N15/8509 , C12N2800/30 , C12N2800/90 , C12N2840/203
摘要： Disclosed herein are compositions and methods for deleting or duplicating DNA in a mammalian genome. Also disclosed are compositions and methods for generating a random genome-wide chromosome rearrangement. Also disclosed are compositions and methods for streamlined construction of gene targeting vectors.
摘要翻译：本文公开了在哺乳动物基因组中缺失或复制DNA的组合物和方法。还公开了用于产生随机全基因组染色体重排的组合物和方法。还公开了用于基因靶向载体的简化构建的组合物和方法。

3. 发明申请

WO2009018018A2 ANIMAL MODEL OF SYNOVIAL SARCOMA 审中-公开
标题翻译： SYNOVIAL SARCOMA的动物模型
公开(公告)号：WO2009018018A2
公开(公告)日：2009-02-05
申请号：PCT/US2008/070769
申请日：2008-07-22
申请人： UNIVERSITY OF UTAH RESEARCH FOUNDATION , HALDAR, Malay , CAPECCHI, Mario, R.
发明人： HALDAR, Malay , CAPECCHI, Mario, R.
IPC分类号： A01K67/027
CPC分类号： C07K14/82 , A01K67/0275 , A01K2217/052 , A01K2217/206 , A01K2227/105 , A01K2267/0331 , A01K2267/0393 , C07K2319/00 , C12N15/8509
摘要： Synovial sarcoma is an aggressive soft-tissue malignancy. Disclosed herein is an animal model of synovial sarcoma wherein one or more myogenic cells of the animal express recombinant SYT-SSX fusion polypeptide. Using this model, myoblasts were identified as a source of synovial sarcoma. Remarkably, within the skeletal muscle lineage, while expression of the oncoprotein in immature myoblasts leads to induction of synovial sarcoma with 100% penetrance, its expression in more differentiated cells induces myopathy without tumor induction. In addition, early widespread expression of the disclosed fusion protein disrupts normal embryogenesis, causing lethality.
摘要翻译：滑膜肉瘤是一种侵袭性软组织恶性肿瘤。本文公开了滑膜肉瘤的动物模型，其中动物的一种或多种肌原细胞表达重组SYT-SSX融合多肽。使用该模型，成肌细胞被鉴定为滑膜肉瘤的来源。值得注意的是，在骨骼肌谱系中，尽管未成熟成肌细胞中癌蛋白的表达导致诱导具有100％外显率的滑膜肉瘤，但其在更多分化细胞中的表达诱导肌病而没有肿瘤诱导。另外，所公开的融合蛋白的早期普遍表达破坏了正常的胚胎发生，导致致死性。

4. 发明申请

WO2002096470A1 A POPULATION OF HOXB8 MUTANTS AND USES IN IDENTIFYING AGENTS FOR TREATING REPETITIVE BEHAVIORS 审中-公开
标题翻译： HOXB8突变体的种群和用于鉴定代谢行为的鉴定剂
公开(公告)号：WO2002096470A1
公开(公告)日：2002-12-05
申请号：PCT/US2002/017611
申请日：2002-05-31
申请人： UNIVERSITY OF UTAH RESEARCH FOUNDATION , GREER, Joy, M. , CAPECCHI, Mario, R.
发明人： GREER, Joy, M. , CAPECCHI, Mario, R.
IPC分类号： A61K49/00
CPC分类号： A61K49/0008
摘要： This invention provides in vivo and in vitro methods of screening for an agent or a combination of agents that reduces one or more repetitive behaviors, comprising contacting neuronal cells of an animal with a HOXB8 gene mutation with the agent or combination of agents to be screened, and determining whether one or more repetitive behaviors of the animal is reduced or whether one or more biochemical correlates of repetitive behaviors is reduced, the reduction in one or more repetitive behaviors or biochemical correlates indicating an agent or combination of agents that reduces repetitive behaviors. The invention also provides a method of treating a subject with repetitive behaviors, comprising administering a therapeutically effective dose of the agent or combination of agents identified by the screening method. Further provided is a population of animals with a HOXB8 gene mutation, wherein more than 30% of the animals have excessive grooming behaviors and wherein less than 10% of the animals show skeletal defects.
摘要翻译：本发明提供了体内和体外筛选减少一种或多种重复行为的试剂或组合组合的方法，包括将动物的神经元细胞与HOXB8基因突变与待筛选的试剂或试剂的组合接触，并且确定动物的一个或多个重复行为是否减少，或者一个或多个重复行为的生物化学相关性是否减少，一种或多种重复行为或生物化学相关性的降低指示减轻重复行为的药剂或药剂组合。本发明还提供了一种以重复行为治疗受试者的方法，包括施用治疗有效剂量的该试剂或通过筛选方法鉴定的试剂组合。还提供了具有HOXB8基因突变的动物群体，其中超过30％的动物具有过度的修饰行为，并且其中少于10％的动物显示骨骼缺陷。

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