CRISPR/Cas9系统：一种用于体内和体外基因治疗的强大技术。
CRISPR/Cas9 system: a powerful technology for in vivo and ex vivo gene therapy.
作者信息
Zhang Xiaohui, Wang Liren, Liu Mingyao, Li Dali
机构信息
Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University, Shanghai, 200241, China.
出版信息
Sci China Life Sci. 2017 May;60(5):468-475. doi: 10.1007/s11427-017-9057-2. Epub 2017 Apr 20.
CRISPR/Cas9 is a versatile genome-editing tool which is widely used for modifying the genome of both prokaryotic and eukaryotic organisms for basic research and applications. An increasing number of reports have demonstrated that CRISPR/Cas9-mediated genome editing is a powerful technology for gene therapy. Here, we review the recent advances in CRISPR/Cas9-mediated gene therapy in animal models via different strategies and discuss the challenges as well as future prospects.
CRISPR/Cas9是一种多功能的基因组编辑工具，广泛应用于原核生物和真核生物基因组的修饰，用于基础研究和实际应用。越来越多的报道表明，CRISPR/Cas9介导的基因组编辑是一种用于基因治疗的强大技术。在此，我们综述了通过不同策略在动物模型中CRISPR/Cas9介导的基因治疗的最新进展，并讨论了面临的挑战以及未来前景。
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