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[利用CRISPR/Cas9进行基因组编辑:迈向医学新时代的第一步?]

[Genome Editing with CRISPR/Cas9: First Steps Towards a new Era in Medicine?].

作者信息

Schermer Bernhard, Benzing Thomas

出版信息

Dtsch Med Wochenschr. 2019 Feb;144(4):276-281. doi: 10.1055/a-0759-7180. Epub 2019 Feb 13.

DOI:10.1055/a-0759-7180
PMID:30759475
Abstract

The emergence of genome editing technologies can be regarded as one of the most groundbreaking revolutions in the history of science. Modern genome editing allows the introduction of precise mutations into the genome of virtually all cells and organisms without leaving any additional trace. Undoubtedly, genome editing with CRISPR/Cas9, often casually referred to as "genetic scissors", will revolutionize medical research and development. However, at the same time it creates a great need for ethical considerations as it might hold risks for both people and the environment that cannot yet be fully assessed. While genome editing is already well established in laboratory research, clinical applications based on genome editing are close. For the first time, targeted corrections of genetic defects in somatic cells, stem cells as well as in the germ line appear technically feasible. This generates possible future scenarios that urgently require broad ethical and social discussions.

摘要

基因组编辑技术的出现可被视为科学史上最具开创性的革命之一。现代基因组编辑能够在几乎所有细胞和生物体的基因组中引入精确的突变,且不留下任何额外痕迹。毫无疑问,使用CRISPR/Cas9进行的基因组编辑,常被戏称为“基因剪刀”,将给医学研发带来变革。然而,与此同时,它也引发了对伦理考量的强烈需求,因为它可能对人类和环境都存在尚未能完全评估的风险。虽然基因组编辑在实验室研究中已相当成熟,但基于基因组编辑的临床应用已近在咫尺。首次,对体细胞、干细胞以及生殖系中的基因缺陷进行靶向校正,在技术上似乎可行。这催生了一些可能的未来情景,迫切需要进行广泛的伦理和社会讨论。

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引用本文的文献

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CRISPR-Cas9 Genome Editing in Human Cell Lines with Donor Vector Made by Gibson Assembly.利用 Gibson 组装法制备供体载体对人细胞系进行 CRISPR-Cas9 基因组编辑。
Methods Mol Biol. 2020;2115:365-383. doi: 10.1007/978-1-0716-0290-4_20.