基因和细胞兴奋剂:新的前沿-超越神话或现实。

Medicine and sport science Pub Date : 2017-01-01 Epub Date: 2017-06-02 DOI:10.1159/000465456
Elmo W I Neuberger, Perikles Simon
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引用次数: 17

摘要

临床研究中基因转移技术的出现引起了人们的担忧,即这些技术将被滥用于提高运动成绩的目的。然而,在过去的20年里,基因治疗领域已经走过了漫长而曲折的道路,只有少数基因治疗药物在人类中显示出临床益处。基因治疗的现状是,病毒载体介导的基因转移在临床试验中显示出人们期待已久的初步成功,即安全的,在某些情况下有效的基因转移。此外,使用小干扰RNA有望通过基因沉默进行有效治疗,尽管仍存在一些安全问题。最近,分子生物学CRISPR/Cas9系统的发展为高效和高度靶向的基因组编辑开辟了新的可能性。本章旨在定义并因此揭开“基因兴奋剂”一词的神秘面纱,并讨论目前有关基因和细胞为基础的物理增强策略的现实。阐述了基因治疗领域的技术进展,重点介绍了近年来的临床进展和技术难点。将这些技术的吸引力与传统兴奋剂做法进行比较,可以发现目前的基因治疗技术对兴奋剂目的仍然没有吸引力,不太可能超过传统兴奋剂。然而,未来的技术进步可能会提高基因兴奋剂的吸引力,从而使其更容易制定检测策略。本章介绍了目前可用的检测策略,表明原则上已经可以检测到许多形式的遗传操作。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Gene and Cell Doping: The New Frontier - Beyond Myth or Reality.

The advent of gene transfer technologies in clinical studies aroused concerns that these technologies will be misused for performance-enhancing purposes in sports. However, during the last 2 decades, the field of gene therapy has taken a long and winding road with just a few gene therapeutic drugs demonstrating clinical benefits in humans. The current state of gene therapy is that viral vector-mediated gene transfer shows the now long-awaited initial success for safe, and in some cases efficient, gene transfer in clinical trials. Additionally, the use of small interfering RNA promises an efficient therapy through gene silencing, even though a number of safety concerns remain. More recently, the development of the molecular biological CRISPR/Cas9 system opened new possibilities for efficient and highly targeted genome editing. This chapter aims to define and consequently demystify the term "gene doping" and discuss the current reality concerning gene- and cell-based physical enhancement strategies. The technological progress in the field of gene therapy will be illustrated, and the recent clinical progress as well as technological difficulties will be highlighted. Comparing the attractiveness of these technologies with conventional doping practices reveals that current gene therapy technologies remain unattractive for doping purposes and unlikely to outperform conventional doping. However, future technological advances may raise the attractiveness of gene doping, thus making it easier to develop detection strategies. Currently available detection strategies are introduced in this chapter showing that many forms of genetic manipulation can already be detected in principle.

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