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使用重组DNA技术表达的活性马碳酸化人体因子IX

H de la Salle, W Altenburger, R Elkaim

|July 18, 1985

概括

再组合DNA技术使肝瘤细胞能够产生活跃的人类因子IX. 这一突破为治疗B型血友病,一种严重的出血障碍提供了有希望的途径.

科学领域:

生物技术是生物技术.
分子生物学分子生物学
血液学血液学血液学

背景情况:

第九因子 (圣诞因子) 对于血液凝固至关重要.
缺少第九因子会导致B型血友病,这是一种遗传性出血障碍.
目前的处理涉及到因子IX缩物,需要改进生产方法.

研究的目的:

探索重组DNA技术的使用,以产生功能的人类第九因子.
评估基于疫苗病毒的载体对表达复杂蛋白质的疗效.
为了评估不同细胞类型的因子IX的产生.

主要方法:

一个人类的第九因子cDNA克隆被插入到疫苗病毒载体中.
重组载体被用来感染人类肝瘤细胞和小鼠纤维细胞.
在产生的蛋白质中测量了因子IX活性.

主要成果:

人类肝瘤细胞成功地产生了完全活跃的第九因子.
无论维生素K水平如何,小鼠纤维细胞都会产生一种不那么活跃的因子IX形式.
这证明了复杂的,经过翻译修改的蛋白质通过重组DNA技术的成功表达.

结论:

基于疫苗病毒的载体可以在真核细胞中有效地表达活性,复杂的治疗性蛋白质,如因子IX.
这种方法对大规模生产血友病B治疗的因子IX具有重大潜力.
该研究强调了用于临床应用的重组蛋白质生产的进步.

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