赵钦军¹,韩忠朝²

• 1:北京汉氏联合生物技术股份有限公司
• 2:中国医学科学院血液病医院(血液学研究所)实验血液学国家重点实验室

摘要(Abstract)：

2015年4月我国英文学术期刊《蛋白质与细胞》（Potein and Cell）发表了中山大学黄军就副教授关于利用CRISPR/Cas9基因编辑技术对人类胚胎中地中海贫血症致病基因修饰的研究成果，引起了国内外对基因编辑技术及其应用研究的大讨论。本文简要介绍了几种不同的基因组编辑技术的原理，比较分析了它们的优缺点。由于ZFNs和TALENs相对低效有更容易发生脱靶等缺点，而NgAgo基因编辑技术在几个月之前刚刚发表，目前的研究文献不多，因此本文主要总结了CRISPR/Cas基因编辑技术的研究进展，对其疾病基因治疗的应用前景进行了展望，同时对涉及的伦理和相关法规建设问题进行了分析并对监管机构制定政策提出了有益建议。

关键词(KeyWords)： 基因编辑技术;人类生殖细胞;人类胚胎;伦理;法律法规

Abstract:

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基金项目(Foundation):

作者(Author): 赵钦军¹,韩忠朝²

参考文献(References)：

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