清脆的
核糖核蛋白
基因组编辑
电穿孔
反式激活crRNA
生物
基因组工程
分子生物学
基因传递
同源定向修复
转染
肽
Cas9
引导RNA
细胞生物学
计算生物学
基因
核糖核酸
遗传学
生物化学
DNA修复
DNA错配修复
作者
Dana V. Foss,Joseph J. Muldoon,David N. Nguyen,Daniel J.J. Carr,Srishti U. Sahu,John M. Hunsinger,Stacia K. Wyman,Netravathi Krishnappa,Rima Mendonsa,Elaine V. Schanzer,Brian R. Shy,Vivasvan S. Vykunta,Vincent Allain,Zhongmei Li,Alexander Marson,Justin Eyquem,Ross C. Wilson
标识
DOI:10.1038/s41551-023-01032-2
摘要
CRISPR-mediated genome editing of primary human lymphocytes is typically carried out via electroporation, which can be cytotoxic, cumbersome and costly. Here we show that the yields of edited primary human lymphocytes can be increased substantially by delivering a CRISPR ribonucleoprotein mixed with an amphiphilic peptide identified through screening. We evaluated the performance of this simple delivery method by knocking out genes in T cells, B cells and natural killer cells via the delivery of Cas9 or Cas12a ribonucleoproteins or an adenine base editor. We also show that peptide-mediated ribonucleoprotein delivery paired with an adeno-associated-virus-mediated homology-directed repair template can introduce a chimaeric antigen receptor gene at the T-cell receptor α constant locus, and that the engineered cells display antitumour potency in mice. The method is minimally perturbative, does not require dedicated hardware, and is compatible with multiplexed editing via sequential delivery, which minimizes the risk of genotoxicity. The peptide-mediated intracellular delivery of ribonucleoproteins may facilitate the manufacturing of engineered T cells.
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