Concepts and strategies for human gene therapy.
Concepts and strategies for human gene therapy.
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人类基因治疗的概念和策略。
DOI：
10.1111/j.1432-1033.1992.tb17176.x
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发表时间：
1992
期刊：
影响因子：
--
通讯作者：
Friedmann,T
中科院分区：
文献类型：
--
作者：
Roemer,K;Friedmann,T
现代分子遗传学方法已经发展起来，可以在人类和其他哺乳动物的体细胞中稳定地转移和表达外源DNA序列。因此，这些方法被应用于补充遗传缺陷和纠正疾病表型的尝试也就不足为奇了。二十年的研究现在已经导致了第一次临床适用的尝试，将转基因细胞引入人类，以治愈至少部分由遗传缺陷引起的疾病。我们在这里讨论了在体内应用治疗性基因转移所遵循的一些策略，并总结了与体细胞基因治疗相关的一些技术和概念上的困难。
Methods of modern molecular genetics have been developed that allow stable transfer and expression of foreign DNA sequences in human and other mammalian somatic cells. It is therefore no surprise that the methods have been applied in attempts to complement genetic defects and correct disease phenotypes. Two decades of research have now led to the first clinically applicable attempts to introduce genetically modified cells into human beings to cure diseases caused at least partially by genetic defects. We discuss here some of the strategies being followed for bothin vitroandin vivoapplication of therapeutic gene transfer and summarize some of the technical and conceptual difficulties associated with somatic‐cell gene therapy.