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1. 发明申请

US20130203083A1 SERUM CLUSTERIN LEVELS IN SYSTEMIC AMYLOIDOSIS FEATURING CARDIOMYOPATHY 审中-公开
标题翻译：系统性淀粉样变性细胞因子血清白蛋白水平
公开(公告)号：US20130203083A1
公开(公告)日：2013-08-08
申请号：US13578294
申请日：2011-02-10
申请人： Lawreen Connors , Michael J. Greene
发明人： Lawreen Connors , Michael J. Greene
IPC分类号： G01N33/68
CPC分类号： G01N33/6893 , G01N2333/775
摘要： The present invention relates generally to diagnostic methods, systems, assays and kits for identification of subjects with cardiac amyloid deposits, where a low level of clusterin protein in a peripheral fluid sample, e.g., a serum sample from the subject, indicates the subject likely has cardiac amyloid deposits. Other aspects relate to methods of treatment of diseases or disorders characterized by cardiac amyloid deposits and transthyretin (TTR) amyloidosis, and more particularly to methods of treatment of cardiac-related amyloidosis and cardiac amyloid deposits in subjects with familial transthyretin (TTR), senile systemic amyloidosis (SSA), or familial amyloidodic polyneuropathy (FAP), or immunoglobulin light chain (AL) amyloidosis. Other aspects relate to methods and compositions comprising clusterin (CLU) or a clusterin agent (e.g. an agonist of clusterin activity or a biologically active fragment or derivative thereof), and their use in methods to treat a disease or disorder characterized by transthyretin (TTR) amyloidosis, e.g. senile systemic amyloidosis (SSA) or familial amyloidodic polyneuropathy (FAP), and their use in methods to treat amyloidotic cardiomyopathy associated with transthyretin (TTR) amyloidosis.
摘要翻译：本发明一般涉及用于鉴定具有心脏淀粉样蛋白沉积物的受试者的诊断方法，系统，测定和试剂盒，其中外周液体样品中的低水平的蛋白聚糖蛋白，例如来自受试者的血清样品，表明受试者可能具有心脏淀粉样蛋白沉积其他方面涉及以心脏淀粉样沉积物和转甲状腺素蛋白淀粉样变性（TTR）淀粉样变性为特征的疾病或病症的治疗方法，更具体地涉及治疗家族性甲状腺激素（TTR）的老年性系统性心脏淀粉样变性和心脏淀粉样蛋白沉积的方法淀粉样变性（SSA）或家族性淀粉样多发性神经病（FAP）或免疫球蛋白轻链（AL）淀粉样变性。其它方面涉及包含聚蛋白（CLU）或聚集蛋白剂（例如聚集蛋白活性的激动剂或其生物活性片段或衍生物）的方法和组合物，以及它们在治疗以转甲状腺素蛋白（TTR）为特征的疾病或病症的方法中的用途，淀粉样变性，例如老年性系统性淀粉样变性病（SSA）或家族性淀粉样变性多发性神经病变（FAP），以及它们用于治疗与转甲状腺素蛋白（TTR）淀粉样变性相关的淀粉样变性心肌病的方法。

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