Kaufman R J
Genetics Institute, Cambridge, MA 02140-2387.
Trends Biotechnol. 1991 Oct;9(10):353-9. doi: 10.1016/0167-7799(91)90118-2.
Current therapy for hemophilia A requires frequent infusion of plasma-derived human factor VIII with the associated drawbacks of potential viral contamination, high cost and limited plasma availability. Factor VIII replacement therapy has been improved through increased knowledge of molecular mechanisms regulating blood coagulation, derived largely from the isolation of the factor VIII gene and its expression in mammalian cells. Homogeneous pure preparations of factor VIII--the largest, most complex protein pharmaceutical produced to date through recombinant DNA technology--can now be produced for successful treatment of hemophilia A.
目前治疗甲型血友病的方法需要频繁输注血浆源性人凝血因子VIII，存在潜在病毒污染、成本高和血浆供应有限等相关缺点。通过对调节血液凝固分子机制的深入了解，凝血因子VIII替代疗法得到了改进，这在很大程度上源于凝血因子VIII基因的分离及其在哺乳动物细胞中的表达。现在可以生产出凝血因子VIII的均质纯制剂——这是迄今为止通过重组DNA技术生产的最大、最复杂的蛋白质药物——用于成功治疗甲型血友病。
