GENETIC ENGINEERING AN IMPROVED FACTOR 8 PRODUCT

凝血因子VIII是一种血浆蛋白， 血友病A患者，导致临床出血性疾病。 患有这种疾病的患者需要频繁输注因子VIII， 为了维持止血，特别是在手术期间或手术后 身体创伤 目前，商业因子VIII产品是纯化的， 从人供体获得的合并血浆中。 最近的事态发展 已经使得生产商业上可行量的因子VIII成为可能 使用重组宿主/载体系统。 这种生产的产品 该方案应具有相对于 血浆衍生产物。 这些包括更高的纯度，更低的体积， 显著降低病毒污染的风险。 建议的目的 一项研究计划是对因子VIII蛋白进行遗传修饰， 提高其药理效用，同时降低治疗成本 使用重组DNA技术。

Factor VIII is the plasma protein which is functionally deficient in hemophilia A patients, resulting in the clinical bleeding disorder. Patients with this disease require frequent infusions of factor VIII in order to maintain hemostasis, particularly during surgery or following physical trauma. Presently, the commercial factor VIII product is purified from pooled blood plasma obtained from human donors. Recent developments have made it possible to produce commercially viable amounts of factor VIII using recombinant host/vector systems. The product from such a production scheme should have substantially improved properties relative to the plasma-derived product. These include higher purity, lower volumes and markedly reduced risk of viral contamination. The aim of the proposed research plan is to genetically modify the factor VIII protein to further improve its pharmacologic utility while reducing the cost of treatment using recombinant DNA technologies.