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11. 发明申请

WO2016090276A1 NRAS RELATED CANCER THERAPY 审中-公开
标题翻译： NRAS相关癌症治疗
公开(公告)号：WO2016090276A1
公开(公告)日：2016-06-09
申请号：PCT/US2015/064048
申请日：2015-12-04
申请人： BRANDEIS UNIVERSITY
发明人： REN, Ruibao
IPC分类号： A61P35/00
CPC分类号： C12Q1/48 , A01K67/0275 , A01K2217/052 , A01K2227/105 , A01K2267/0331 , A61K31/513 , A61K45/06 , A61K49/0008 , C12Y203/01 , C12Y306/05002 , G01N33/573 , G01N2333/91057 , G01N2333/914 , G01N2440/10 , G01N2500/04 , G01N2500/10 , G01N2800/7028 , A61K2300/00
摘要： The present invention encompasses the recognition that identification of alternative means to block RAS oncogenic signaling may be required for developing novel cancer therapies. Among other things, the present invention encompasses the recognition that targeting RAS palmitoylation can achieve effective therapy for RAS-related cancers. Furthermore, the present invention encompasses the recognition that reduction of ZDHHC9 level and/or activity can significantly reduce palmitoylation level of Ras protein. Among other things, the present invention encompasses the recognition that identification of agents that modulate expression and/or activity of ZDHHC9 can reduce palmitoylation level of Ras protein. In some embodiments, the present invention provides methods of treating a subject suffering from cancer by administering ZDHHC9 inhibition therapy.
摘要翻译：本发明包括认识到鉴定阻断RAS致癌信号的替代手段可能是开发新型癌症疗法所必需的。除此之外，本发明包括认识到靶向RAS棕榈酰化可以实现RAS相关癌症的有效治疗。此外，本发明包括认识到减少ZDHHC9水平和/或活性可以显着降低Ras蛋白的棕榈酰化水平。除此之外，本发明包括识别调节ZDHHC9的表达和/或活性的试剂可以降低Ras蛋白的棕榈酰化水平。在一些实施方案中，本发明提供了通过施用ZDHHC9抑制疗法治疗患有癌症的受试者的方法。

12. 发明申请

WO2016083856A1 TRANSGENIC MOUSE FOR THE ASSESSMENT OF THYROID HORMONE (TH) ACTION 审中-公开
标题翻译：用于评估甲状腺激素（TH）作用的转基因小鼠
公开(公告)号：WO2016083856A1
公开(公告)日：2016-06-02
申请号：PCT/HU2015/050020
申请日：2015-11-30
申请人： MAGYAR TUDOMÁNYOS AKADÉMIA KÍSÉRLETI ORVOSTUDOMÁNYI KUTATÓ INTÉZET
发明人： FEKETE, Csaba , GEREBEN, Balázs , MOHÁCSIK, Petra , ERDÉLYI, Ferenc , SZABÓ, Gábor
IPC分类号： A01K67/027 , C12N15/85
CPC分类号： A01K67/0275 , A01K2217/052 , A01K2217/07 , A01K2217/203 , A01K2227/105 , A01K2267/0393 , A61K49/0008 , C12N15/85 , C12N15/8509 , C12N2015/859 , C12N2800/90 , C12N2830/002 , C12Q1/6897 , G01N33/5088 , G01N33/78
摘要： A transgenic animal model that is suitable for the cell or tissue specific assessing of thyroid hormone (TH) action in vivo is described. The recombinant DNA construct and methods suitable to generate such an animal are also provided. The assessment of TH action is based on a reporter that is dependent on an endogenously expressed thyroid hormone receptor (TR) and coregulators of said receptor.
摘要翻译：描述了适合于体内甲状腺激素（TH）作用的细胞或组织特异性评估的转基因动物模型。还提供了重组DNA构建体和适于产生这种动物的方法。 TH作用的评估基于依赖于内源性表达的甲状腺激素受体（TR）和所述受体的调节剂的报告物。

13. 发明申请

WO2016059166A1 HUMANIZED NEUROMELANIN-CONTAINING RODENT 审中-公开
标题翻译：含人神经元素的RODENT
公开(公告)号：WO2016059166A1
公开(公告)日：2016-04-21
申请号：PCT/EP2015/073909
申请日：2015-10-15
申请人： FUNDACIÓ HOSPITAL UNIVERSITARI VALL D'HEBRON - INSTITUT DE RECERCA , INSTITUCIÓ CATALANA DE RECERCA I ESTUDIS AVANÇATS , CENTRO DE INVESTIGACIÓN BIOMÉDICA EN RED ENFERMEDADES NEURODEGENERATIVAS (CIBERNED)
发明人： VILA BOVER, Miquel , CARBALLO CARBAJAL, Iria , BOVÉ BADELL, Jordi , CUADROS ARASA, Thais , LAGUNA TUSET, Ariadna
IPC分类号： A01K67/027 , C12N9/02 , A61K49/00
CPC分类号： A61K49/0008 , A01K67/0278 , A01K2227/105 , A01K2267/0318 , C12N9/0059 , C12N2750/14143 , C12N2830/003 , C12N2830/008
摘要： The invention provides a humanized rodent exhibiting age-dependent production and intracellular accumulation of human-like neuromelanin, up to levels reached in elder humans, within Parkinson's disease-vulnerable neuronal groups. The humanized neuromelanin-containing rodent stably expresses the human tyrosinase gene in at least the dopaminergic neurons of the substantia nigra pars compacta and can be a wild-type rodent to which the human tyrosinase gene has been transduced via a viral vector, or a transgenic rodent that expresses the human gene in a constitutive or inducible form. In any of these cases, the rodent develops features of Parkinson's disease such as nigro-striatal dopaminergic neurodegeneration and motor impairment, making it a valuable tool for the (i) search of biomarkers for the early detection, diagnosis or progression of the disease, and (ii) the screening of new therapies with disease-modifying potential to combat PD.
摘要翻译：本发明提供了在帕金森氏病易感神经元组中，人类化的啮齿动物在老年人中达到的人类样神经丝氨酸的年龄依赖性产生和细胞内积累。含有人源化神经丝氨酸的啮齿动物在至少黑质黑质多巴胺能神经元中稳定地表达人酪氨酸酶基因，并且可以是通过病毒载体或转基因啮齿类动物转导人酪氨酸酶基因的野生型啮齿动物其以组成型或诱导型形式表达人基因。在任何这些情况下，啮齿动物发展出帕金森病的特征，例如黑质纹状体多巴胺能神经变性和运动障碍，使其成为（i）搜索生物标志物以用于疾病的早期发现，诊断或进展的有价值的工具，以及（ii）筛选具有改善疾病的潜力的新疗法以对抗PD。

14. 发明申请

WO2016036424A1 ASSAY, COMPOSITION AND NON-ENZYMATIC MECHANISM OF STATIN IN MODULATING LIPID METABOLISM 审中-公开
标题翻译：调节脂质代谢中的染料的测定，组成和非酶解机理
公开(公告)号：WO2016036424A1
公开(公告)日：2016-03-10
申请号：PCT/US2015/027704
申请日：2015-04-27
申请人： PLAXGEN
发明人： MADASAMY, Shanmugavel
IPC分类号： G01N33/92
CPC分类号： G01N33/92 , A61K49/0008 , G01N33/94 , G01N2500/02
摘要： A novel non-enzymatic assay for lipid modulating drugs is being described for biological samples. For the first time non-enzymatic mechanism of statin drugs in modulating lipid aggregates and forming lipid particles is being shown. A simple flow cytometer based testing for detection of lipid particles, the effect of lipid modulating drug such as statin in vitro and testing for efficacy of the drug for an individual, dosage adjustment and drug discovery mechanism is being described. The lipid modulating drugs either individually or in combination can be discovered, optimized or efficacy tested using this assay. A novel statin induced lipid particle formation described can be used for diagnosis, personalized medicine and biomarker identification as well.
摘要翻译：正在描述一种用于脂质调节药物的新型非酶分析用于生物样品。正在显示他汀类药物调节脂质聚集体和形成脂质颗粒的非酶促机制。正在描述基于用于脂质颗粒检测的简单流式细胞仪测试，脂质调节药物如他汀类药物在体外的作用以及药物对个体的功效的测试，剂量调整和药物发现机制。单独或组合的脂质调节药物可以使用该测定法发现，优化或功效测试。描述的新型他汀类药物诱导的脂质颗粒形成也可用于诊断，个性化医学和生物标志物鉴定。

15. 发明申请

WO2015196114A2 COMPOSITIONS AND METHODS FOR MODULATING NEURONAL DEGENERATION 审中-公开
标题翻译：用于调节神经元变性的组合物和方法
公开(公告)号：WO2015196114A2
公开(公告)日：2015-12-23
申请号：PCT/US2015036755
申请日：2015-06-19
申请人： UNIV ARKANSAS
发明人： KIAEI MAHMOUD
IPC分类号： A01K67/027 , C12N15/85
CPC分类号： C07K14/4716 , A01K67/0275 , A01K67/0278 , A01K2207/15 , A01K2217/052 , A01K2217/072 , A01K2217/206 , A01K2227/105 , A01K2267/0318 , A61K49/0008 , C12N15/8509
摘要： The present disclosure provides genetically modified animals and cells comprising a polynucleotide encoding human profilin1. Also provided are methods of assessing the effects of agents in genetically modified animals and cells comprising a polynucleotide encoding human profilin1.
摘要翻译：本公开提供了转基因动物和细胞，其包含编码人类雌激素1的多核苷酸。还提供了评价试剂在转基因动物和细胞中的作用的方法，所述细胞包含编码人类profilin1的多核苷酸。

16. 发明申请

WO2015195768A1 HUMANIZED MOUSE MODEL AND USES THEREOF 审中-公开
标题翻译：人造鼠模型及其用途
公开(公告)号：WO2015195768A1
公开(公告)日：2015-12-23
申请号：PCT/US2015/036180
申请日：2015-06-17
申请人： CHAMPIONS BIOTECHNOLOGY, INC.
发明人： BAIA, Gilson, S. , PAZ, Keren , SIDRANSKY, David
IPC分类号： A61K49/00
CPC分类号： A01K67/0271 , A01K2207/12 , A01K2207/15 , A01K2227/105 , A01K2267/03 , A01K2267/0331 , A61K49/0008
摘要： The invention relates generally to a humanized mouse model and uses thereof. Specifically, the invention relates to methods for generating, maintaining, and expanding a culture of leukocytes in heterologous animals. This invention also relates to use of these animals as models of human immune system for testing molecules in order to treat a disease or disorder such as cancer.
摘要翻译：本发明一般涉及人源化小鼠模型及其用途。具体地说，本发明涉及产生，维持和扩大异源动物白细胞培养的方法。本发明还涉及这些动物作为人类免疫系统的模型来测试分子以便治疗诸如癌症的疾病或病症的用途。

17. 发明申请

WO2015193706A1 METHOD FOR ALZHEIMER'S DISEASE DRUG SCREENING AND BIOMARKER EVALUATION 审中-公开
标题翻译：阿尔茨海默病药物筛选和生物标记评估方法
公开(公告)号：WO2015193706A1
公开(公告)日：2015-12-23
申请号：PCT/IB2014/062497
申请日：2014-06-20
申请人： FUNDACIÓN FRAUNHOFER CHILE RESEARCH
发明人： DEACON, Robert , PEREZ-COGRAM, Patricia , SCHUCH, Wolfgang
IPC分类号： A61K49/00
CPC分类号： A61K49/0008 , A01K2227/10 , A01K2267/0312
摘要： Octogon degus are the only rodents known to spontaneously develop an Alzheimer's disease (AD)-like neuropathology with hallmark pathological markers, such as amyloid-β (Αβ) plaques and tau hyperphosphorylation, and a concomitant age-related decline in cognition. Nevertheless, it is difficult to discriminate between healthy animals, and those developing the AD-like disease. The present invention is directed to identify individuals that will likely develop AD-like neuropathology. Also, the invention considers a method for drug screening of potentially beneficial compounds, and a method for identification of AD biomarkers.
摘要翻译： Octogon degus是唯一已知可以自发发展阿尔茨海默病（AD）样神经病理学的具有标志性病理学标志物的诸如淀粉样蛋白-β（Αβ）斑块和tau高度磷酸化的啮齿类动物，以及伴随的年龄相关的认知衰退。然而，难以区分健康动物和发展AD样疾病的动物。本发明旨在鉴定将可能开发AD样神经病理学的个体。此外，本发明还考虑了潜在有益化合物的药物筛选方法，以及鉴定AD生物标志物的方法。

18. 发明申请

WO2015132670A3 METHODS AND COMPOSITIONS FOR DETECTION OF TARGETS FOR AUTOIMMUNE DISEASE 审中-公开
标题翻译：用于检测自身免疫疾病目标的方法和组合物
公开(公告)号：WO2015132670A3
公开(公告)日：2015-12-23
申请号：PCT/IB2015000912
申请日：2015-03-07
申请人： UNIV HEALTH NETWORK
发明人： HERSHENFIELD BRIAN , MAK TAK W
IPC分类号： C40B30/06 , C12N15/113 , C12Q1/00 , C12Q1/68 , C40B30/04 , C40B40/06 , G01N33/48 , G01N33/483
CPC分类号： C12Q1/6883 , A61K38/177 , A61K39/39541 , A61K39/39558 , A61K49/0008 , A61K2039/505 , A61K2039/507 , C07K14/70503 , C07K16/2818 , C07K2317/76 , C12N15/1135 , C12N15/1138 , C12N2310/11 , C12N2310/14 , C12N2320/30 , C12Q1/6886 , C12Q2600/106 , C12Q2600/158 , G01N33/5088 , G01N2333/82 , A61K2300/00
摘要： The present invention is directed to methods for identifying one or more target genes that modulate the activation state of dendritic cells. The methods are based on transfection of a siRNA library into immature bone marrow-derived dendritic cells and detecting the activation state of said cells. The identified one or more target genes can be further validated as candidate targets for induction of self-tolerance and treatment of autoimmune diseases.
摘要翻译：本发明涉及用于鉴定调节树突状细胞的活化状态的一种或多种靶基因的方法。所述方法基于将siRNA文库转染到未成熟骨髓来源的树突状细胞中并检测所述细胞的活化状态。所鉴定的一个或多个靶基因可以进一步验证为诱导自身免疫性疾病的自我耐受和治疗的候选靶标。

19. 发明申请

WO2015185977A1 ANIMAL MODELS OF CORNEAL ANGIOGENESIS AND CORNEAL ECTATIC DISEASES, METHODS OF PRODUCING, AND METHODS OF USE THEREOF 审中-公开
标题翻译：脊柱角质瘤和角膜疾病的动物模型，生产方法及其使用方法
公开(公告)号：WO2015185977A1
公开(公告)日：2015-12-10
申请号：PCT/IB2015/000822
申请日：2015-06-03
申请人： SOLIS HERRERA, Arturo
发明人： SOLIS HERRERA, Arturo
IPC分类号： A01N37/34
CPC分类号： A01K67/027 , A01K2227/10 , A01K2267/035 , A61K31/03 , A61K31/05 , A61K49/0008
摘要： Methods of producing non-human animal models of corneal angiogenesis and corneal ectatic diseases, such as corneal keratoconus, by applying an aromatic compound to the eye of a non-human animal are described. Also described are non-human animal models of corneal angiogenesis and corneal ectatic diseases, and methods of using the non-human animal models to screen compounds that modulate corneal angiogenesis and corneal ectatic diseases.
摘要翻译：描述了通过将非芳香化合物施用于非人类动物的眼睛来产生角膜血管生成和角膜外膜疾病如角膜圆锥角膜的非人动物模型的方法。还描述了角膜血管生成和角膜外膜疾病的非人动物模型，以及使用非人动物模型筛选调节角膜血管生成和角膜外膜疾病的化合物的方法。

20. 发明申请

WO2015179317A2 GENETICALLY MODIFIED NON-HUMAN ANIMALS EXPRESSING HUMAN EPO 审中-公开
标题翻译：基因改造非人类动物表达人类EPO
公开(公告)号：WO2015179317A2
公开(公告)日：2015-11-26
申请号：PCT/US2015031429
申请日：2015-05-18
申请人： REGENERON PHARMA , UNIV YALE , INST RES IN BIOMEDICINE IRB
发明人： MURPHY ANDREW J , STEVENS SEAN , FLAVELL RICHARD , MANZ MARKUS , SHAN LIANG
IPC分类号： C12N7/00
CPC分类号： A01K67/0271 , A01K67/0276 , A01K67/0278 , A01K2207/12 , A01K2207/15 , A01K2217/05 , A01K2217/072 , A01K2217/075 , A01K2217/15 , A01K2227/105 , A01K2267/01 , A01K2267/0337 , A01K2267/0381 , A61K49/0008 , C07K14/4703 , C07K14/475 , C07K14/505 , C07K14/524 , C07K14/535 , C07K14/5403 , C07K14/5437 , C07K14/7155 , C12N9/00 , C12N9/14 , C12Q1/18 , C12Y301/03048 , G01N33/5088
摘要： Genetically modified non-human animals expressing human EPO from the animal genome are provided. Also provided are methods for making non-human animals expressing human EPO from the non-human animal genome, and methods for using non-human animals expressing human EPO from the non-human animal genome. These animals and methods find many uses in the art, including, for example, in modeling human erythropoiesis and erythrocyte function; in modeling human pathogen infection of erythrocytes; in in vivo screens for agents that modulate erythropoiesis and/or erythrocyte function, e.g. in a healthy or a diseased state; in in vivo screens for agents that are toxic to erythrocytes or erythrocyte progenitors; in in vivo screens for agents that prevent against, mitigate, or reverse the toxic effects of toxic agents on erythrocytes or erythrocyte progenitors; in in vivo screens of erythrocytes or erythrocyte progenitors from an individual to predict the responsiveness of an individual to a disease therapy.
摘要翻译：提供了从动物基因组表达人EPO的转基因非人动物。还提供了从非人动物基因组制备表达人EPO的非人动物的方法，以及从非人动物基因组中使用表达人EPO的非人动物的方法。这些动物和方法在本领域中发现许多用途，包括例如对人红细胞生成和红细胞功能的建模; 在模拟人类病原体感染红细胞; 在用于调节红细胞生成和/或红细胞功能的药剂的体内筛选中。处于健康或疾病状态; 在用于对红细胞或红细胞祖细胞有毒的药剂的体内筛选中; 在体内筛选用于防止，减轻或逆转毒性剂对红细胞或红细胞祖细胞的毒性作用的药剂; 在体内筛选来自个体的红细胞或红细胞祖细胞以预测个体对疾病治疗的反应性。

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