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首页> 外文期刊>Proceedings of the National Academy of Sciences of the United States of America >Gene therapy for the hemophilias
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Gene therapy for the hemophilias

机译：血友病的基因治疗

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摘要

The hemophilias are a group of severe bleeding disorders resulting from the lack of functional blood coagulation factorⅧ (hemophilia A) or factor Ⅸ (hemophilia B). Factor Ⅸ, when activated, catalyzes the conversion of factor Ⅹ to its activated form, and activated factor Ⅷ serves as a cofactor in this reaction. Clinically the disorders are indistinguishable, and they were not distinguished in the laboratory until the 1950s, when two research teams independently demonstrated that plasma from one group of patients could correct the clotting defect of the other group (1, 2). Clinically the disease is characterized by frequent spontaneous bleeds into joints and soft tissues, with the potential to result in a chronic and debilitating arthropathy. More serious complications, and death, can result from bleeding into other critical closed spaces, such as the intracranial or the retroperito-neal space. The disease is classified as severe, moderate, or mild, based on circulating-factor levels, with severe defined as < 1% of normal activity, moderate as 1-5%, and mild as 5-30%.
机译：血友病是由于缺乏功能性凝血因子Ⅷ（血友病A）或凝血因子Ⅸ（血友病B）导致的一组严重出血性疾病。当活化时，因子Ⅸ催化因子Ⅹ转化为其活化形式，并且活化的因子Ⅷ在该反应中用作辅因子。从临床上讲，这种疾病是无法区分的，直到1950年代才在实验室中加以区分，当时两个研究小组独立地证明一组患者的血浆可以纠正另一组患者的凝血缺陷（1、2）。临床上，该疾病的特征是频繁自发地渗入关节和软组织，并有可能导致慢性和使人衰弱的关节炎。更严重的并发症和死亡可能是由于出血进入其他关键的封闭空间，例如颅内或腹膜后间隙。根据循环因子水平，该疾病分为严重，中度或轻度，重度定义为正常活动的<1％，中度为1-5％，轻度为5-30％。

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