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[基因治疗及其应用]

[Gene therapy and its applications].

作者信息

Austin-Ward E D, Villaseca C

机构信息

Departamento de Medicina, Hospital Clínico Universidad de Chile, Santiago, Chile.

出版信息

Rev Med Chil. 1998 Jul;126(7):838-45.

PMID:9830778
Abstract

Gene therapy consists in the introduction of genes in cells of the human organism. It allows the treatment of not only monogenic diseases (those in which one gene is altered, such as rare enzymatic diseases), but also of diseases in which there is an acquired gene alteration such as AIDS and cancer, and diseases in which several genes and the environment interact, such as diabetes and coronary artery disease. In the few years of development of this therapy several clinical trials have been approved, that, along with experiment of this therapy several clinical trials have been approved, that, along with experimental research, are offering promising results. In this new field of modern medicine, specially in experiments related to genetic manipulation of germinal cells, ethical aspects have great importance.

摘要

基因治疗是指将基因导入人体细胞。它不仅可以治疗单基因疾病(即一个基因发生改变的疾病,如罕见的酶缺乏症),还可以治疗获得性基因改变的疾病,如艾滋病和癌症,以及几种基因与环境相互作用的疾病,如糖尿病和冠状动脉疾病。在这种治疗方法发展的短短几年里,已经有多项临床试验获得批准,这些试验与实验研究一起都取得了令人鼓舞的结果。在现代医学的这个新领域,特别是在与生殖细胞基因操作相关的实验中,伦理方面非常重要。

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