Gene therapy for inborn errors of immunity: Base editing comes into play

被引：3

作者：

Malech, Harry L. ^{[1
]}

Notarangelo, Luigi D. ^{[1
]}

机构：

[1] NIH, Natl Inst Allergy & Infect Dis, Lab Clin Immunol & Microbiol, Bethesda, MD 20892 USA

来源：

CELL | 2023年 / 186卷 / 07期

基金：

美国国家卫生研究院;

关键词：

GENERATION;

D O I：

10.1016/j.cell.2023.03.001

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

CRISPR-Cas9-based base editing allows precise base editing to achieve conversion of adenosine to guanine or cytosine to thymidine. In this issue of Cell, McAuley et al. use adenine base editing to correct a single base-pair mutation causing human CD3d deficiency, demonstrating superior efficiency of genetic correction with reduced undesired genetic alterations compared with standard CRISPR-Cas9 editing.

引用

页码：1302 / 1304

页数：3

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