Progress toward human gene therapy.
Progress toward human gene therapy.
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DOI：
10.1097/00006254-199007000-00004
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发表时间：
1989-06
期刊：
影响因子：
56.9
通讯作者：
T. Friedmann
中科院分区：
文献类型：
--
作者：
T. Friedmann
目前对大多数人类遗传疾病的治疗是不够的。为了满足对有效治疗的需求，现代分子遗传学正在通过直接攻击突变基因为前所未有的疾病治疗新方法提供工具。最近的结果与几个靶器官和基因转移技术已导致广泛的医学和科学接受的可行性，这种“基因治疗”的概念，对疾病的骨髓，肝脏和中枢神经系统;某些种类的癌症;和缺乏循环酶，激素和凝血因子。最完善的模式涉及通过重组致病病毒的基因转移改变突变靶基因，以表达新的遗传信息并纠正疾病表型-将病理学的剑转化为治疗的犁头。
Current therapies for most human genetic diseases are inadequate. In response to the need for effective treatments, modern molecular genetics is providing tools for an unprecedented new approach to disease treatment through an attack directly on mutant genes. Recent results with several target organs and gene transfer techniques have led to broad medical and scientific acceptance of the feasibility of this "gene therapy" concept for disorders of the bone marrow, liver, and central nervous system; some kinds of cancer; and deficiencies of circulating enzymes, hormones, and coagulation factors. The most well-developed models involve alteration of mutant target genes by gene transfer with recombinant pathogenic viruses in order to express new genetic information and to correct disease phenotypes--the conversion of the swords of pathology into the plowshares of therapy.