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21. 发明申请

WO02059150A3 MATERIALS AND METHODS RELATING TO PROTEIN AGGREGATION IN NEURODEGENERATIVE DISEASE 审中-公开
标题翻译：相关蛋白质聚集在神经病变疾病中的材料与方法
公开(公告)号：WO02059150A3
公开(公告)日：2002-12-05
申请号：PCT/GB0200005
申请日：2002-01-02
申请人： UNIV ABERDEEN , WISCHIK CLAUDE MICHEL , RICKARD JANET ELIZABETH , HORSLEY DAVID , HARRINGTON CHARLES ROBERT , THEURING FRANZ , STAMER KARSTEN , ZABKE CLAUDIA
发明人： WISCHIK CLAUDE MICHEL , RICKARD JANET ELIZABETH , HORSLEY DAVID , HARRINGTON CHARLES ROBERT , THEURING FRANZ , STAMER KARSTEN , ZABKE CLAUDIA
IPC分类号： A01K67/027 , A61K45/00 , A61P25/28 , A61P43/00 , C07K14/47 , C07K19/00 , C12N5/10 , C12N15/09 , C12N15/12 , C12N15/85 , C12P21/02 , G01N33/15 , G01N33/50 , G01N33/53 , G01N33/68 , C12N15/11 , C12N15/62
CPC分类号： C12N15/8509 , A01K67/0275 , A01K2207/15 , A01K2217/00 , A01K2217/05 , A01K2217/20 , A01K2227/105 , A01K2267/0312 , A01K2267/0318 , C07K14/4711 , C07K2319/00 , G01N33/6896 , G01N2500/00
摘要： Disclosed are methods of inducing or modelling the pathological state of an aggregating disease protein (ADP - e.g. tau protein) which is associated with a disease state in which the ADP aggregates pathologically (e.g. Alzheimer's disease) through an induced conformational polymerisation interaction, the method being characterised by the step of providing a membrane-localisable fusion protein comprising (i) an aggregating portion, which is derived from the ADP, or from a protein which initiates pathological aggregation of the ADP, (ii) a heterologous membrane-localising portion. Membrane-localisation of the ADP-based fusion protein is believed to cause the high-affinity capture site of the ADP protein to become exposed such that aggregation of further ADP, which may be native or heterologous to the system, to be promoted. The method can be carried out in vitro, or in cell- and animal-models, and may be used to screen for modulators of the aggregation process by monitoring aggregation e.g. by monitoring the production of the ADP-related degradation products resulting from the aggregation. Also provided are materials, processes for use in or with the methods.
摘要翻译：公开了诱导或模拟与通过诱导的构象聚合相互作用在ADP聚集物（例如阿尔茨海默病）的疾病状态相关的聚集性疾病蛋白（ADP-例如tau蛋白）的病理状态的方法，所述方法是其特征在于提供可膜可定位的融合蛋白的步骤，其包含（i）衍生自ADP的聚集部分或引发ADP的病理性聚集的蛋白质，（ii）异源膜定位部分。认为基于ADP的融合蛋白的膜定位导致ADP蛋白的高亲和力捕获位点被暴露，使得可以促进对系统天然或异源的另外的ADP的聚集。该方法可以在体外或细胞和动物模型中进行，并且可以用于通过监测聚集来筛选聚集过程的调节剂。通过监测由聚合产生的与ADP相关的降解产物的产生。还提供了用于或与该方法一起使用的材料，方法。

22. 发明申请

WO0226107A3 MODEL FOR NEURODEGENERATIVE DISEASES INVOLVING AMYLOID ACCUMULATION 审中-公开
标题翻译：涉及AMYLOID积累的神经病变疾病模型
公开(公告)号：WO0226107A3
公开(公告)日：2002-08-22
申请号：PCT/US0129788
申请日：2001-09-25
申请人： UNIV CALIFORNIA
发明人： LYNCH GARY , XIAONING BI , GALL CHRISTINE M
IPC分类号： C07K14/775 , C12N15/85 , C12Q1/00 , C12Q1/68 , G01N33/50 , G01N33/68 , A01K67/00 , A01K67/033 , A01N43/04 , A61K31/70 , A61K39/00 , A61K39/38 , C07H19/00 , C07H21/00 , C12N5/00 , C12N5/02 , C12N15/00 , C12N15/09 , C12N15/63 , C12N15/70 , C12N15/74 , C12N15/87 , G01N33/00 , G01N33/53 , G01N33/567
CPC分类号： C07K14/775 , A01K2207/15 , A01K2217/00 , A01K2217/05 , A01K2217/075 , A01K2227/105 , A01K2267/0312 , A01K2267/0318 , C12N15/8509 , G01N33/5058 , G01N33/6896 , G01N2800/52
摘要： The present invention provides brain cells, such as normal brain cells, apolipoprotein E deficient brain cells, or apoE4 containing brain cells, that are treated with a compound which can modulate integrins and/or integrin receptors to produce increased sequestration of and/or accumulation of and/or uptake of A beta , and/or changed in cathepsin D content and/or lysosomal dysfunction, and/or microglia activation in the brain cells. The present invention also provides methods for producing such cells and methods for using the cells for screening an agent or substance that modulates the sequestration of and/or accumulation of and/or uptake of A beta , and/or lysosomal dysfunction, and/or changes in cathepsin D content and/or microglia activation in the brain cells. The method further provides a new therapeutic target, antagonism of glutamate receptors, for the treatment of neurodegenerative diseases which are characterized by inter alia , abnormal amyloid uptake and/or accumulation .
摘要翻译：本发明提供脑细胞，例如正常脑细胞，载脂蛋白E缺乏型脑细胞或含有apoE4的脑细胞，其可用可调节整联蛋白和/或整联蛋白受体的化合物治疗以产生增加的聚集和/或累积的和/或摄取Aβ，和/或改变组织蛋白酶D含量和/或溶酶体功能障碍，和/或脑细胞中的小胶质细胞激活。本发明还提供了用于产生这种细胞的方法以及使用该细胞筛选调节Aβ和/或溶酶体功能障碍的螯合和/或积累和/或摄取和/或变化的试剂或物质的方法在脑细胞中的组织蛋白酶D含量和/或小胶质细胞激活。该方法进一步提供了治疗神经变性疾病的新的治疗靶标，谷氨酸受体的拮抗作用，其特征在于特异性淀粉样蛋白摄取和/或积累。

23. 发明申请

WO0236736A3 ALPHA-TOCOPHEROL TRANSFER PROTEIN KNOCKOUT ANIMALS 审中-公开
标题翻译： ALPHA-生育酚转移蛋白敲除动物
公开(公告)号：WO0236736A3
公开(公告)日：2002-08-08
申请号：PCT/US0145249
申请日：2001-11-01
申请人： UNIV CALIFORNIA , FARESE JR ROBERT V , TERASAWA YUKO , TRABER MARET G
发明人： FARESE JR ROBERT V , TERASAWA YUKO , TRABER MARET G
IPC分类号： C07K14/47 , C12N15/85 , A01K67/00 , G01N33/00
CPC分类号： C12N15/8509 , A01K67/0276 , A01K2217/072 , A01K2217/075 , A01K2227/105 , A01K2267/03 , A01K2267/0318 , A01K2267/0331 , A01K2267/0356 , A01K2267/0375 , C07K14/47 , C12N2800/30
摘要： This invention provides knockout animals comprising a disruption in one or both alleles of the gene encoding alpha-tocopherol transfer protein (TTP). The knockout animals provide good model systems for atherosclerosis.
摘要翻译：本发明提供了包含编码α-生育酚转运蛋白（TTP）的基因的一个或两个等位基因中的破坏的敲除动物。敲除动物为动脉粥样硬化提供了良好的模型系统。

24. 发明申请

WO2002059381A2 GENE FOR IDENTIFYING INDIVIDUALS WITH FAMILIAL DYSAUTONOMIA 审中-公开
标题翻译：用家族性遗传识别个体的基因
公开(公告)号：WO2002059381A2
公开(公告)日：2002-08-01
申请号：PCT/US2002/000473
申请日：2002-01-07
申请人： THE GENERAL HOSPITAL CORPORATION , SLAUGENHAUPT, Susan , GUSELLA, James, F.
发明人： SLAUGENHAUPT, Susan , GUSELLA, James, F.
IPC分类号： C12Q1/68
CPC分类号： C12Q1/6883 , A01K2217/05 , A01K2217/075 , A01K2227/105 , A01K2267/0306 , A01K2267/0318 , C07K14/47 , C12N15/8509 , C12Q2600/156
摘要： This invention relates to methods and compositions useful for detecting mutations which cause Familial Dysautonomia. Familial dysautonomia (FD; Riley-Day syndrome), an Ashkenazi Jewish disorder, is the best known and most frequent of a group of congenital sensory neuropathies and is characterized by widespread sensory and variable autonomic dysfunction. Previously, we mapped the FD gene, DYS , to a 0.5 cM region of chromosome 9q31 and showed that the ethnic bias is due to a founder effect, with >99.5% of disease alleles sharing a common ancestral haplotype. To investigate the molecular basis of FD, we sequenced the minimal candidate region and cloned and characterized its 5 genes. One of these, IKBKAP , harbors two mutations that can cause FD. The major haplotype mutation is located in the donor splice site of intron 20. This mutation can result in skipping of exon 20 in the mRNA from FD patients, although they continue to express varying levels of wild-type message in a tissue-specific manner. RNA isolated from patient lymphoblasts is primarily wild-type, whereas only the deleted message is seen in RNA isolated from brain. The mutation associated with the minor haplotype in four patients is a missense (R696P) mutation in exon 19 that is predicted to disrupt a potential phosphorylation site. Our findings indicate that almost all cases of FD are caused by an unusual splice defect that displays tissue-specific expression; and they also provide the basis for rapid carrier screening in the Ashkenazi Jewish population.
摘要翻译：本发明涉及可用于检测引起家族性躯体障碍的突变的方法和组合物。家族性失眠症（FD; Riley-Day综合征），一种Ashkenazi犹太病症，是一组先天性感觉神经病最知名和最常见的，其特征在于广泛的感觉和可变自主神经功能障碍。以前，我们将FD基因DYS映射到染色体9q31的0.5cM区域，并显示种族偏倚是由于创始者效应，> 99.5％的疾病等位基因共享共同的祖先单体型。为了研究FD的分子基础，我们对最小候选区进行了测序，克隆并表征了其5个基因。其中之一，IKBKAP，有两个可能导致FD的突变。主要的单倍型突变位于内含子20的供体剪接位点。这种突变可导致FD患者mRNA中外显子20的跳跃，尽管它们以组织特异性方式继续表达不同程度的野生型信息。从患者淋巴母细胞分离的RNA主要是野生型，而只有从脑分离的RNA中才能看到已删除的信息。与4例患者中较小单倍型相关的突变是外显子19中的错义（R696P）突变，预测会破坏潜在的磷酸化位点。我们的研究结果表明，几乎所有FD的病例都是由显示组织特异性表达的不寻常的拼接缺陷引起的; 它们也为在阿什肯纳西犹太人群体中快速的载体筛查提供了基础。

25. 发明申请

WO02026107A2 MODEL FOR NEURODEGENERATIVE DISEASES INVOLVING AMYLOID ACCUMULATION 审中-公开
标题翻译：涉及AMYLOID积累的神经病变疾病模型
公开(公告)号：WO02026107A2
公开(公告)日：2002-04-04
申请号：PCT/US2001/029788
申请日：2001-09-25
IPC分类号： C07K14/775 , C12N15/85 , C12Q1/00 , C12Q1/68 , G01N33/50 , G01N33/68 , A61B
CPC分类号： C07K14/775 , A01K2207/15 , A01K2217/00 , A01K2217/05 , A01K2217/075 , A01K2227/105 , A01K2267/0312 , A01K2267/0318 , C12N15/8509 , G01N33/5058 , G01N33/6896 , G01N2800/52
摘要： The present invention provides brain cells, such as normal brain cells, apolipoprotein E deficient brain cells, or apoE4 containing brain cells, that are treated with a compound which can modulate integrins and/or integrin receptors to produce increased sequestration of and/or accumulation of and/or uptake of A beta , and/or changed in cathepsin D content and/or lysosomal dysfunction, and/or microglia activation in the brain cells. The present invention also provides methods for producing such cells and methods for using the cells for screening an agent or substance that modulates the sequestration of and/or accumulation of and/or uptake of A beta , and/or lysosomal dysfunction, and/or changes in cathepsin D content and/or microglia activation in the brain cells. The method further provides a new therapeutic target, antagonism of glutamate receptors, for the treatment of neurodegenerative diseases which are characterized by inter alia , abnormal amyloid uptake and/or accumulation.
摘要翻译：本发明提供脑细胞，例如正常脑细胞，载脂蛋白E缺乏型脑细胞或含有apoE4的脑细胞，其可用可调节整联蛋白和/或整联蛋白受体的化合物治疗以产生增加的聚集和/或累积的和/或摄取Aβ，和/或改变组织蛋白酶D含量和/或溶酶体功能障碍，和/或脑细胞中的小胶质细胞激活。本发明还提供了用于产生这种细胞的方法以及使用该细胞筛选调节Aβ和/或溶酶体功能障碍的螯合和/或积累和/或摄取和/或变化的试剂或物质的方法在脑细胞中的组织蛋白酶D含量和/或小胶质细胞激活。该方法进一步提供新的治疗靶标，谷氨酸受体的拮抗作用，用于治疗特征在于异常淀粉样蛋白摄取和/或累积的神经变性疾病。

26. 发明申请

WO02024868A2 NON-HUMAN MODEL OF GESTATIONAL AND ADULT FOLATE DEFICIENCY AND USES THEREOF 审中-公开
标题翻译：非人类和成人畸形的非人格模型缺陷及其用途
公开(公告)号：WO02024868A2
公开(公告)日：2002-03-28
申请号：PCT/US2001/029966
申请日：2001-09-24
IPC分类号： C07K14/705 , C12N15/85 , G01N33/50 , C12N
CPC分类号： A01K67/0278 , A01K67/0276 , A01K2207/15 , A01K2217/00 , A01K2217/05 , A01K2217/075 , A01K2227/105 , A01K2267/03 , A01K2267/0306 , A01K2267/0312 , A01K2267/0318 , A01K2267/0331 , A01K2267/0375 , C07K14/705 , C12N15/8509 , G01N33/5008 , G01N33/5011
摘要： Disclosed is a genetically modified non-human animal that is a model for defective folate transport. Specifically the non-human animal comprises a genetic modification that results in decreased or absent expression and/or biological activity of the endogenous reduced folate carrier. The animal can be further modified by introduction into the genome of a nucleid acid molecule encoding the human reduced folate carrier. Such an animal can be used in methods to identify compounds useful for treating a variety of conditions associated with defective folate transport, or for the identification of anti-folate compounds useful for treating cancer. The animal can also be used to study the biochemical and molecular events associated with folate transport. Compounds identified using such a model are valuable for therapeutic treatments.
摘要翻译：公开了一种遗传修饰的非人类动物，其是有缺陷的叶酸运输的模型。具体地说，非人类动物包括导致内源性还原叶酸载体表达和/或生物活性降低或缺失的遗传修饰。可以通过将编码人类还原叶酸载体的核酸分子引入基因组中进一步修饰动物。这样的动物可以用于鉴定可用于治疗与有缺陷的叶酸运输相关的各种病症的化合物的方法，或用于鉴定可用于治疗癌症的抗叶酸化合物。该动物也可用于研究与叶酸运输相关的生物化学和分子事件。使用这种模型鉴定的化合物对于治疗性治疗是有价值的。

27. 发明申请

WO0188109A2 MODEL FOR NEURODEGENERATIVE DISEASE 审中-公开
标题翻译：神经病变疾病模型
公开(公告)号：WO0188109A2
公开(公告)日：2001-11-22
申请号：PCT/GB0102218
申请日：2001-05-18
申请人： CONSEJO SUPERIOR INVESTIGACION , RUFFLES GRAHAM KEITH , LOZANO JOSE JAVIER LUCAS , PEREZ FELIX HERNANDEZ , GRADO JESUS AVILA DE
发明人： PEREZ FELIX HERNANDEZ , DE GRADO JESUS AVILA , LOZANO JOSE JAVIER LUCAS
IPC分类号： A01K67/027 , C12N9/12 , C12N15/09 , C12N15/85 , C12N9/00
CPC分类号： C12N15/8509 , A01K67/0275 , A01K2207/15 , A01K2217/00 , A01K2217/05 , A01K2217/20 , A01K2227/105 , A01K2267/0312 , A01K2267/0318 , C12N9/1205
摘要： A transgenic animal in which GSK-3 beta protein is over-expressed is useful as a model for neurodegenerative disease.
摘要翻译：其中GSK-3β蛋白过度表达的转基因动物可用作神经变性疾病的模型。

28. 发明申请

WO0116176A3 TRANSGENIC ANIMAL MODEL FOR NEURODEGENERATIVE DISEASES 审中-公开
标题翻译：神经退行性疾病的转基因动物模型
公开(公告)号：WO0116176A3
公开(公告)日：2001-09-27
申请号：PCT/EP0008071
申请日：2000-08-18
申请人： BIOFRONTERA PHARMACEUTICALS AG , LUEBBERT HERMANN
发明人： LUEBBERT HERMANN
IPC分类号： A01K67/02 , A01K67/027 , A61K49/00 , C07K14/47 , C12N1/15 , C12N1/19 , C12N1/21 , C12N5/10 , C12N9/00 , C12N15/09 , C12N15/12 , C12N15/85 , C12Q1/68
CPC分类号： C12N9/93 , A01K2207/15 , A01K2217/00 , A01K2217/05 , A01K2217/075 , A01K2267/03 , A01K2267/0318 , A61K49/0008 , C12N15/8509
摘要： The present application refers to a mouse parkin2 DNA- and protein sequence, containing mutations or deletions causing Parkinson's disease in a human if occurring in the according human sequence, the construction of a transgenic non-human animal containing such a mutated DNA sequence and therefore expressing no or a less active or non-active parkin protein as well as the use of this transgenic animal as a model for neurodegenerative diseases.
摘要翻译：本申请涉及小鼠parkin2DNA-和蛋白质序列，其包含在相应的人序列中发生的在人中引起帕金森氏病的突变或缺失，构建含有这种突变的DNA序列的转基因非人动物并因此表达没有或具有较低活性或无活性的parkin蛋白以及使用该转基因动物作为神经退行性疾病的模型。

29. 发明申请

WO01060794A2 METHOD FOR SCREENING FOR ANTI-AMYLOIDOGENIC PROPERTIES AND METHOD FOR TREATMENT OF NEURODEGENERATIVE DISEASE 审中-公开
标题翻译：用于筛选抗肿瘤物质的方法和治疗神经病变疾病的方法
公开(公告)号：WO01060794A2
公开(公告)日：2001-08-23
申请号：PCT/US2001/005569
申请日：2001-02-20
IPC分类号： A01K67/027 , A61K38/00 , A61K48/00 , C07K14/47 , C12N15/85 , C07D
CPC分类号： C12N15/8509 , A01K67/0275 , A01K67/0276 , A01K67/0278 , A01K2207/15 , A01K2217/00 , A01K2217/05 , A01K2217/075 , A01K2227/105 , A01K2267/0312 , A01K2267/0318 , A61K38/00 , A61K48/00 , C07K14/47 , C07K14/4711 , C12N2830/008
摘要： The methodologies of the present invention demonstrate that a critical balance between pro- and anti-amyloidogenic molecules exists that regulates amyloid formation and cell death in Alzheimer's disease and Parkinson's disease. beta -Synuclein, the non-amyloidogenic homologue of alpha -synuclein, is a negative modulator of alpha -synuclein and A beta aggregation, having neuroprotective properties against alpha -synuclein and A beta neurotoxicity and that beta -synuclein and therapeutic agents derived therefrom block amyloidogenesis and neurodegeneration in vivo . The method of the present invention establishes that beta -synuclein blocks A beta aggregation either by direct inhibition of A beta amyloidogenesis or indirectly via either alpha -synuclein or its 35 a.a. NAC region, inferring neuroprotective characteristics within the effected cells. The generation of a transgenic mouse line and a cell-free system overexpressing alpha -synuclein characterizes the mechanisms by which beta -synuclein blocks alpha -synuclein and A beta aggregation and that this mechanism offers protection to the cell against amyloid formation as seen in the pathologies of Alzheimer's disease and Parkinson's disease.
摘要翻译：本发明的方法证明，存在调节淀粉样蛋白形成和阿尔茨海默病和帕金森病中的细胞死亡的促淀粉样变性分子和抗淀粉样蛋白形成分子之间的关键平衡。 β-突触核蛋白是α-突触核蛋白的非淀粉样变性同源物，是α-突触核蛋白和Aβ聚集的负调节剂，具有针对α-突触核蛋白和Aββ神经毒性的神经保护性质，并且β-突触核蛋白和衍生自其的治疗剂阻断淀粉样变性和体内神经变性。本发明的方法确定β-突触核蛋白通过直接抑制Aβ淀粉样蛋白发生或间接通过α-突触核蛋白或其αααα阻断Aβ聚集。 NAC区域，推断受影响细胞内的神经保护特征。转基因小鼠系和过表达α-突触核蛋白的无细胞系统的产生表征β-突触核蛋白阻断α-突触核蛋白和Aβ聚集的机制，并且该机制为细胞免受淀粉样蛋白形成提供保护，如在病理学中所见的阿尔茨海默病和帕金森病。

30. 发明申请

WO01013715A2 ANIMAL MODEL AND USES THEREOF 审中-公开
标题翻译：动物模型及其用途
公开(公告)号：WO01013715A2
公开(公告)日：2001-03-01
申请号：PCT/GB2000/003274
申请日：2000-08-23
IPC分类号： C07K14/705 , C12N15/85 , A01K67/027
CPC分类号： C12N15/8509 , A01K67/0276 , A01K2217/075 , A01K2227/105 , A01K2267/03 , A01K2267/0318 , C07K14/705
摘要： Animal models are provided for pathologies involving monoamine dysfunction in which the vesicular monoamine transporter (2) ( VMAT2 ) gene has been manipulated. Such models are useful particularly in study of various disorders, especially Parkinson's disease, schizophrenia and drug dependencies, and in assay methods for obtaining agents of therapeutic potential in such disorders. Assays and methods of generating animal models are also provided.
摘要翻译：提供动物模型用于涉及单胺功能障碍的病态，其中已经操纵了囊泡单胺转运蛋白（2）（VMAT2）基因。这些模型特别用于研究各种疾病，特别是帕金森病，精神分裂症和药物依赖性，以及用于获得在这种疾病中具有治疗潜力的试剂的测定方法。还提供了产生动物模型的测定和方法。

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