图7:CBE系统的分类(Cheng et al. 2019)CBE 系统的活性窗口定义为 sgRNA 中编辑效率>40%的C位点。(a)前置型CBE(FSCBEs),其活性窗口主要位于sgRNA靶位点的前端(PAM位点看作最末端)。(b)后置型 CBE(BSCBEs),活性窗口主要位于 sgRNA 靶位点的后端。(c)广谱型 CBE(BRCBEs),活性窗口横跨 sgRNA 靶位点的前后端。
单碱基编辑技术有着相当广泛的应用(图8),它在动物模型的构建、疾病治疗、作物育种以及点突变筛选等等多个方面都极具潜力 [13]。开发安全性高、适用范围广的单碱基编辑工具将有助于其潜力的兑现,从而更好的服务于生物医学研究。新的 CBE 工具在安全性和适用范围上均取得了相当大的进步,为单碱基编辑技术的进步做出了贡献。
图8 单碱基编辑技术的应用前景 (改自K.A.Molla et al. 2019)
作者简介
程田林博士,助理研究员,中国科学院脑科学与智能技术卓越创新中心,中国科学院神经科学研究所
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