Gene Therapy: An Outstanding Technique For Diseases

Nayade Sarmiento, S. Tinoco
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Abstract

Nearly three decades ago, the first clinical trial based on gene therapy revolutionized the scientific field by demonstrating that it was possible to genetically modify harmful or defective cells and therapeutically improve patient conditions. Since then, this technique has been implemented for the study and treatment of different diseases, many of them fatal, with the hope of achieving a "cure". Several genetic editing tools have been developed and continue to be studied to improve the effectiveness of gene therapy, in addition to a more exhaustive analysis for choosing the type of vector, which is the main cause of adverse effects. In this review, we present characteristics of the gene therapy mechanism along with the types of vectors that are used in this procedure, followed by the most important applications in the medical field and briefly we describe some limitations and prospects to study in the future.
基因治疗:一项杰出的疾病治疗技术
近30年前,第一个基于基因疗法的临床试验彻底改变了科学领域,它证明了对有害或有缺陷的细胞进行基因修饰并从治疗上改善患者状况是可能的。从那时起,这项技术已被用于研究和治疗各种疾病,其中许多是致命的,以期实现“治愈”。为了提高基因治疗的有效性,已经开发并继续研究了几种基因编辑工具,此外还对选择载体类型进行了更详尽的分析,这是产生不良反应的主要原因。在本文中,我们介绍了基因治疗机制的特点以及在此过程中使用的载体类型,其次是在医学领域的最重要的应用,并简要描述了一些局限性和未来研究的前景。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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