PURE FACTOR IX FOR TREATMENT OF HEMOPHILIA B

用于治疗 B 型血友病的 PURE IX 因子

• 批准号: 3501163
• 负责人: DAVID H BING
• 金额: $ 5万
• 依托单位: IMMUNE DISEASE INSTITUTE, INC.
• 依托单位国家: 美国
• 财政年份: 1987
• 资助国家: 美国
• 起止时间: 1987-07-01 至 1988-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/3501163
• 关键词: affinity chromatography; antibody; antibody specificity; antiseptic sterilization; biological products; biotechnology; blood coagulation tests; blood toxicology; blood transfusion; coagulation factor IX; conformation; congenital blood protein disorder; disease /disorder model; divalent cations; dosage; enzyme linked immunosorbent assay; gel electrophoresis; heat; hemophilia B; hepatitis B virus group; high performance liquid chromatography; human immunodeficiency virus 1; human tissue; intravenous administration; microorganism classification; plasma; radioimmunoassay; radiotracer; thermostability; virus classification; zymogens

Hemophilia B is a severe bleeding disorder characterized by a functional deficiency in the blood coagulation protein factor IX (Christmas factor). Therapy for bleeding episodes in these patients requires the intravenous replacement of factor IX given as fresh frozen plasma or with commercially available concentrates of factor IX in which the protein is only partially purified. Although these commercial materials have proven efficacy in this disease, they contain many impurities, are often contaminated with virus, and their use is associated with a significant morbidity. The goal of this project is the development of a highly purified form of factor IX. The factor IX will be obtained by using a recently described invention which employs conformation-specific antibodies against human factor IX to purify this protein. This factor IX will be homogeneous and thus suitable for intravenous therapy. Published studies of factor IX purified by this methodology indicate that it is free of contaminants and infectious agents responsible for complications associated with the currently commercially available concentrates. In this Phase I study sufficient factor IX will be purified to accomplish studies of acute toxicity in rabbits and the ability to correct the bleeding deficiency in hemophiliac dogs.

血友病B是一种严重的出血障碍，特征在 血液凝血蛋白因子IX的功能缺乏 （圣诞节因素）。 这些疗法的出血情节 患者需要静脉替代给定因子IX 作为新鲜的冷冻等离子体或可商购的 蛋白质仅部分的蛋白质IX浓度IX 纯化。 尽管这些商业材料已证明 在这种疾病中的功效，它们含有许多杂质，通常是 被病毒污染，它们的使用与 明显的发病率。 该项目的目的是开发 高度纯化的因子IX。 第IX因素将是 通过使用最近描述的发明获得的 针对人类因素IX的构象特异性抗体 纯化该蛋白质。 这个因素IX将是同质的，因此 适用于静脉治疗。 因素IX的发表研究 通过这种方法纯化表明它没有 负责并发症的污染物和传染剂 与当前可用的 浓缩物。 在这个阶段，我研究了足够的因素IX将是 纯化以完成兔子急性毒性的研究 能够纠正血友病犬出血缺乏症。