CRISPR 起始缺失：通过碱基编辑诱导的起始密码子突变进行基因沉默的一种新颖实用的替代方法。CRISPR Start-Loss: A Novel and Practical Alternative for Gene Silencing through Base-Editing-Induced Start Codon Mutations.-医云文献数字医云科研云海量医学决策数据服务

Abstract：

CRISPR-Cas9-mediated gene knockout and base-editing-associated induction of STOP codons (iSTOP) have been widely used to exterminate the function of a coding gene, while they have been reported to exhibit side effects. In this study, we propose a novel and practical alternative method referred to as CRISPR Start-Loss (CRISPR-SL), which eliminates gene expression by utilizing both adenine base editors (ABEs) and cytidine base editors (CBEs) to disrupt the initiation codon (ATG). CRISPR-SL has been verified to be a feasible strategy on the cellular and embryonic levels (mean editing efficiencies up to 30.67% and 73.50%, respectively) and in two rabbit models mimicking Otc deficiency (Otc gene) and long hair economic traits (Fgf5 gene).

摘要：

CRISPR-Cas9介导的基因敲除和碱基编辑相关的STOP密码子诱导（iSTOP）已被广泛用于消除编码基因的功能，虽然据报道它们有副作用。在这项研究中,我们提出了一种新颖实用的替代方法，称为CRISPR起始损失(CRISPR-SL)，通过利用腺嘌呤碱基编辑器（ABE）和胞苷碱基编辑器（CBE）破坏起始密码子（ATG）来消除基因表达。CRISPR-SL已被验证为在细胞和胚胎水平上的可行策略（平均编辑效率高达30.67％和73.50％，分别）和在两个模拟Otc缺乏症（Otc基因）和长发经济性状（Fgf5基因）的兔模型中。