素数(序理论)
计算机科学
计算生物学
定向进化
限制
蛋白质工程
折叠(DSP实现)
合成生物学
蛋白质表达
理论(学习稳定性)
生物
离体
基因组编辑
表达式(计算机科学)
HEK 293细胞
信使核糖核酸
逆向工程
定向分子进化
氨基酸
理论计算机科学
西格玛
作者
Y. Allen Tao,Holt A. Sakai,Allen Y. Jiang,Nicholas A. Krasnow,Vasilii S. Vaganov,Brian Shim,Zachary Barsdale,Smriti Pandey,Nouraiz Ahmed,Man Na,Ting‐Wei Liao,Keyede Oye,Ana Cristian,Emily Zhang,Joy A. Xu,Mattijs Bulcaen,David R. Liu
标识
DOI:10.1038/s41587-026-03149-6
摘要
Although protein engineering and laboratory evolution have been used to optimize prime editors, we show that previous changes that improve prime editor efficiency also compromise protein stability and expression level, limiting performance. To address these limitations, we apply structure-informed artificial intelligence-guided methods such as the inverse-folding network ProteinMPNN to redesign the reverse transcriptase (RT) domains of engineered and evolved prime editors while preserving regions essential for catalysis. Redesigned RTs are extensively mutated, with 30-163 amino acid substitutions, and exhibit enhanced folding stability and soluble expression and up to twofold higher intracellular prime editor protein levels following mRNA delivery. Redesigned PE8 prime editors demonstrate enhanced editing efficiencies across multiple ex vivo contexts, including in several human primary cell types and via several delivery modalities. In mice, editing efficiency is up to 2.9-fold higher than that of state-of-the-art PE6, PE7 and PEmax prime editors. These findings demonstrate a generalizable approach for augmenting laboratory evolution to improve genome editing agents.
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