AI-guided redesign of laboratory-evolved reverse transcriptases enhances prime editing

Переработка лабораторно эволюционировавших обратных транскриптаз с помощью методов ИИ повышает эффективность prime-редактирования
Holt A. Sakai, Smriti Pandey, Nouraiz Ahmed, Keyede Oye, Ting‐Wei Liao, Emily Zhang, Allen Y. Jiang, Y. Allen Tao, Nicholas A. Krasnow, Vasilii S. Vaganov, Brian Shim, Zachary Barsdale, Man Na, Ana Cristian, Joy A. Xu, Mattijs Bulcaen, David R. Liu
2026-05-21

ProteinMPNNlaboratory evolution augmentationprime editingprotein stability and expressionreverse transcriptase redesign
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.
1
Applied structure-informed AI (ProteinMPNN) to redesign RT domains while preserving catalytic regions.
2
In mice, PE8 editing efficiency is up to 2.9-fold higher than state-of-the-art PE6, PE7, and PEmax editors.
3
Previous RT changes that improve prime editor efficiency can reduce protein stability and expression, limiting performance.
4
Redesigned PE8 editors show enhanced editing efficiencies across multiple ex vivo contexts, human primary cell types, and delivery modalities.
5
Redesigned RTs contain extensive mutations (30–163 amino acid substitutions) and show enhanced folding stability and soluble expression.
6
Redesigned RTs yield up to twofold higher intracellular prime editor protein levels following mRNA delivery.
7
The approach demonstrates a generalizable method to augment laboratory evolution for improving genome editing agents.

Laboratory-evolved reverse transcriptase (RT) domains within engineered prime editors

AI-guided, structure-informed redesign effects on RTs: extensive sequence substitutions that preserve catalytic regions while enhancing folding stability, soluble expression, intracellular prime editor protein levels, and prime editing efficiency across cell types, delivery modalities, and in vivo (mouse) comparisons to PE6/PE7/PEmax

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2026-05-21
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Authors
Holt A. Sakai
Smriti Pandey
Nouraiz Ahmed
Keyede Oye
Ting‐Wei Liao
Emily Zhang
Allen Y. Jiang
Y. Allen Tao
Nicholas A. Krasnow
Vasilii S. Vaganov
Brian Shim
Zachary Barsdale
Man Na
Ana Cristian
Joy A. Xu
Mattijs Bulcaen
David R. Liu
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