CRISPR/Cas9 基因编辑：研究技术、临床应用及伦理考量。
CRISPR/Cas9 gene-editing: Research technologies, clinical applications and ethical considerations.
机构信息
Department of Cell and Developmental Biology, University College London, 21 University Street, WC1E 6DE London, UK.
Yale Cardiovascular Research Center, Section of Cardiovascular Medicine, Yale School of Medicine, 300 George, 7(th) Floor, New Haven, CT 06511, United States.
出版信息
Semin Perinatol. 2018 Dec;42(8):487-500. doi: 10.1053/j.semperi.2018.09.003. Epub 2018 Oct 2.
Gene therapy carries the potential to treat more than 10,000 human monogenic diseases and benefit an even greater number of complex polygenic conditions. The repurposing of CRISPR/Cas9, an ancient bacterial immune defense system, into a gene-editing technology has armed researchers with a revolutionary tool for gene therapy. However, as the breadth of research and clinical applications of this technology continues to expand, outstanding technical challenges and ethical considerations will need to be addressed before clinical applications become commonplace. Here, we review CRISPR/Cas9 technology and discuss its benefits and limitations in research and the clinical context, as well as ethical considerations surrounding the use of CRISPR gene editing.
基因治疗有可能治疗超过 10000 种人类单基因疾病，并使更多复杂的多基因疾病受益。CRISPR/Cas9 的重新利用，一种古老的细菌免疫防御系统，已经为基因治疗的研究人员提供了一个革命性的基因编辑工具。然而，随着这项技术的研究和临床应用的不断扩展，在临床应用变得普遍之前，还需要解决突出的技术挑战和伦理问题。在这里，我们回顾了 CRISPR/Cas9 技术，并讨论了它在研究和临床环境中的益处和局限性，以及围绕 CRISPR 基因编辑使用的伦理问题。
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