血红蛋白病的未来治疗前景。
Future prospects for treatment of hemoglobinopathies.
作者信息
Stamatoyannopoulos J A
机构信息
University of Washington School of Medicine, Seattle.
出版信息
West J Med. 1992 Dec;157(6):631-6.
Abstract
Strategies for the treatment of sickle cell anemia and beta-thalassemia are founded on the knowledge that these disorders result from structural or functional defects in an adult gene for which an intact fetal counterpart exists. During the past decade, several pharmacologic agents have been investigated for their potential to ameliorate sickle cell anemia and beta-thalassemia by increasing the synthesis of fetal hemoglobin in adults. Progress in understanding globin gene regulation is now being combined with advances in retrovirus-mediated gene transfer, and the once-distant goal of providing gene therapy for hemoglobinopathies is rapidly approaching reality.
摘要
镰状细胞贫血和β地中海贫血的治疗策略基于这样的认识：这些疾病是由成人基因的结构或功能缺陷导致的，而胎儿期存在完整的对应基因。在过去十年中，人们研究了几种药物，看它们是否有潜力通过增加成人胎儿血红蛋白的合成来改善镰状细胞贫血和β地中海贫血。目前，对珠蛋白基因调控的理解进展正与逆转录病毒介导的基因转移技术进步相结合，曾经遥不可及的为血红蛋白病提供基因治疗的目标正迅速成为现实。
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本文引用的文献
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