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GENE THERAPY AND GENE TARGETING

机译：基因治疗和基因定位

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摘要

There are about 4000 inherited human diseases known today. The better known of these are : Cystic fibrosis, thalassemias (hemoglobin deficiency), sickle cell anemia, phenylketonuria (amino acid metabolism disorder), citrullinema (deficiency of urea-cycle enzyme argininosuccinate synthetase), Fanconi anemia, Bloom-syndrome (defects in DNA repair), Lesch-Nyhan syndrome (defects in purine metabolism), Hurler syndrome (defect in polysaccharide metabolism) etc. Only few of them are currently treatable. These are caused by defective heritable genes. In most of these diseases the missing defective gene product cannot be supplied exogenously, as insulin can be supplied to diabetics. This is because most enzymes are unstable and cell membrane is impermeable to large molecules of proteins. For most of the inherited diseases, theoretically at least, gene therapy offers the most promising approach to successful treatment. The concept of gene therapy evolved over the last two decades. The principle is simple. A genetic disease is caused by a defective gene resulting from mutation of a given gene. The genetic treatment of such disease can be envisaged by 'replacing' the mutant gene with a normal active copy of it. This replacement can be achieved either by : 1) replacement of its activity by gene complementation or by 2) replacement of the defective gene itself, which is known as gene correction.
机译：今天已知有约4000种遗传的人类疾病。其中更广为人知的是：囊性纤维化，地中海贫血（血红蛋白缺乏症），镰状细胞性贫血，苯酮尿症（氨基酸代谢紊乱），瓜氨酸瘤（尿素循环酶精氨酸琥珀酸酯合成酶缺乏症），范可尼贫血，布鲁姆综合征（DNA缺陷）修复），Lesch-Nyhan综合征（嘌呤代谢缺陷），Hurler综合征（多糖代谢缺陷）等。目前只有少数几种可以治疗。这些是由有缺陷的遗传基因引起的。在大多数这些疾病中，由于胰岛素可以提供给糖尿病患者，因此不能外源提供缺少的缺陷基因产物。这是因为大多数酶不稳定，并且细胞膜不能渗透大分子蛋白质。对于大多数遗传性疾病，至少在理论上，基因治疗为成功治疗提供了最有希望的方法。基因治疗的概念在过去的二十年中得到了发展。原理很简单。遗传疾病是由给定基因突变导致的缺陷基因引起的。这种疾病的遗传治疗可以通过用正常的活性拷贝“替代”突变基因来实现。可以通过以下方式实现这种替换：1）通过基因互补来替换其活性，或者通过2）替换有缺陷的基因本身，这被称为基因校正。

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  • 来源
    《Science and Culture》 |2003年第6期|p.200-202|共3页
  • 作者

    SIBDAS GHOSH;

  • 作者单位

    Centre of Advanced Study, Department of Botany, University of Calcutta, Kolkata-700 019;

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  • 原文格式 PDF
  • 正文语种 eng
  • 中图分类 自然科学总论;
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