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Gene Therapy: An Overview of Current Techniques

机译:基因疗法:当前技术概述

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"Gene therapy" is generally thought of as a very new concept, but the idea that genes could be manipulated to treat human disease goes back several decades. The basic concepts and methodologies for manipulating genes have been taken directly from studies of procaryotic organisms and the viruses that infect them. The idea that viral genomes could become a permanent part of cell genomes was first discovered with bacterial viruses around 1956, and later extended to include animal viruses. One of the earliest mammalian gene transfer experiments was performed by W. Szybalski in 1962. Szybalski developed cell lines that carried mutations in genes responsible for salvage of purine bases and showed that DNA from normal cells could be introduced into these mutant cells to restore the normal phenotype. However, there was little interest in their results because at that time procaryotes, DNA synthesis, and the genetic code were the centers of attention. In 1966, E.L.Tatum gave a talk in New York and covered several aspects of gene therapy including the long-range concept that viruses could be used effectively for man's benefit and that genes would be isolated or synthesized and introduced into defective cells of particular organs. He also speculated that since the basis of cancer is altered genes, treatment could be achieved "by modification and regulation of gene activities or by means of gene repair or replacement." By the late 1960s and early 1970s, gene therapy became the subject of an increasing number of articles and meetings.
机译:人们普遍认为“基因疗法”是一个非常新的概念,但是可以操纵基因来治疗人类疾病的想法可以追溯到几十年前。操纵基因的基本概念和方法已直接从对原核生物及其感染病毒的研究中获得。病毒基因组可以成为细胞基因组永久部分的想法最早是在1956年左右由细菌病毒发现的,后来扩展到包括动物病毒。最早的哺乳动物基因转移实验之一是由W. Szybalski于1962年进行的。Szybalski开发了细胞系,该细胞系携带着负责回收嘌呤碱基的基因中的突变,并表明正常细胞的DNA可以引入这些突变细胞中以恢复正常表型。但是,他们对结果的兴趣不大,因为当时的原核生物,DNA合成和遗传密码是关注的焦点。 1966年,E.L。Tatum在纽约进行了演讲,涵盖了基因治疗的多个方面,其中包括长期概念,即病毒可以有效地用于人类的利益,并且可以分离或合成基因并将其引入特定器官的缺陷细胞中。他还推测,由于癌症的基础是基因改变,因此可以“通过改变和调节基因活性或通过基因修复或置换来实现治疗”。到1960年代末和1970年代初,基因治疗已成为越来越多的文章和会议的主题。

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