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[基因治疗:技术、策略与应用]

[Gene therapy: technics, strategy and application].

作者信息

Hallez S

机构信息

Fondation Y.-Boël, Laboratoire de Chimie biologique, Université Libre de Bruxelles (ULB), Rhode-Saint-Genèse, Belgique.

出版信息

Rev Mal Respir. 1995;12(6):567-83.

PMID:8677351
Abstract

The concept of gene therapy extends to all treatments involving modification of cellular genetics. This approach has numerous applications such as the treatment of genetic disorders, cancer and viral diseases. The first of these implies the introduction of a normal gene to replace the function of the defective gene. In the other two, several strategies may lead to a therapeutic effect. The transfer of genes is equally applicable in any disease where the expression of the gene in the particular tissue is more effective than systemic or local treatment with the corresponding protein (for example Dopamine or Tyrosine hydroxylase for Parkinson's Disease). According to its application and to the strategy chosen, therapeutic gene may be transferred, in vitro or in vivo, with the aid of plasmid vectors or recombinant viruses. These vectors may contain targeting systems and/or regulation of the specific expression of the target cell. Some encouraging results have been obtained for different applications in animals and there are numerous clinical studies currently in progress.

摘要

基因治疗的概念涵盖了所有涉及细胞遗传学修饰的治疗方法。这种方法有许多应用,如治疗遗传疾病、癌症和病毒性疾病。其中第一种意味着引入一个正常基因来替代有缺陷基因的功能。在另外两种情况中,有几种策略可能会产生治疗效果。基因转移同样适用于任何一种疾病,在这种疾病中,特定组织中基因的表达比用相应蛋白质进行全身或局部治疗(例如帕金森病用多巴胺或酪氨酸羟化酶)更有效。根据其应用和所选择的策略,治疗性基因可以借助质粒载体或重组病毒在体外或体内进行转移。这些载体可能包含靶向系统和/或靶细胞特异性表达的调控。在动物的不同应用中已经取得了一些令人鼓舞的结果,目前有许多临床研究正在进行中。

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