CRISPR/Cas9系统应用于早期胚胎编辑和基因治疗

被引：5

作者：

张晓宇 ^{[1
,2
]}

唐蔚 ^{[1
]}

李劲松 ^{[1
,2
]}

机构：

[1] 中国科学院生物化学与细胞生物学研究所分子细胞卓越中心

[2] 细胞生物学国家重点实验室

来源：

生命科学 | 2018年 / 30卷 / 09期

关键词：

CRISPR/Cas9; 胚胎编辑; 基因编辑; 基因治疗;

D O I：

10.13376/j.cbls/2018110

中图分类号：

R450 []; Q78 [基因工程（遗传工程）];

学科分类号：

100215 ; 071007 ; 0836 ; 090102 ;

摘要：

CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats/CRISPR associated protein 9)系统具有高效、精确、操作难度较低等特点,是目前应用最为广泛的基因编辑工具。CRISPR/Cas9在构建模式动物突变体研究基因功能和构建人类疾病模型中有大量使用,但是由于相关伦理限制,在人类胚胎中进行基因编辑来探索治疗性胚胎编辑和研究人早期胚胎发育机制的研究发展较慢。人类胚胎发育机制与小鼠等模式动物的早期胚胎发育机制存在较大差异,过往在模式动物上研究的早期胚胎发育机制要在人类胚胎水平进行验证,对人类胚胎利用基因编辑工具进行直接研究越发必要,探索人类早期胚胎发育机制有助于相关发育疾病的研究与治疗。胚胎治疗性基因编辑可以实现对遗传疾病的彻底阻断,目前还处于初步探索时期,但是主要存在潜在脱靶和编辑后形成嵌合体两方面问题。相较而言,出生后个体水平的基因治疗目前发展较为快速,对遗传疾病和艾滋病等血液疾病有应用价值。现对CRISPR/Cas9近年来应用于人类胚胎编辑和基因治疗的内容进行了综述。

引用

页码：916 / 925

页数：10

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