Gene therapy for inborn errors of immunity: Base editing comes into play.
Cell, 186(7): 1302-1304 PMID 37001495 DOI 10.1016/j.cell.2023.03.001
Аннотация
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 CD3δ deficiency, demonstrating superior efficiency of genetic correction with reduced undesired genetic alterations compared with standard CRISPR-Cas9 editing.