Small RNA Toxin‐Assisted Evolution of GC‐Preferred ErCas12a for Enhanced Genome Targeting Range

Abstract CRISPR/Cas12a, a promising gene editing technology, faces limitations due to its requirement for a thymine (T)‐rich protospacer adjacent motif (PAM). Despite the development of Cas12a variants with expanded PAM profiles, many genomic loci, especially those with guanine‐cytosine (GC)‐rich PA...

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Main Authors: Zehua Chen, Junyuan Xue, Ziying Wang, Jinyuan Sun, Yinglu Cui, Tong Zhu, Huaiyi Yang, Ming Li, Bian Wu
Format: Article
Language:English
Published: Wiley 2025-08-01
Series:Advanced Science
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Online Access:https://doi.org/10.1002/advs.202417105
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author Zehua Chen
Junyuan Xue
Ziying Wang
Jinyuan Sun
Yinglu Cui
Tong Zhu
Huaiyi Yang
Ming Li
Bian Wu
author_facet Zehua Chen
Junyuan Xue
Ziying Wang
Jinyuan Sun
Yinglu Cui
Tong Zhu
Huaiyi Yang
Ming Li
Bian Wu
author_sort Zehua Chen
collection DOAJ
description Abstract CRISPR/Cas12a, a promising gene editing technology, faces limitations due to its requirement for a thymine (T)‐rich protospacer adjacent motif (PAM). Despite the development of Cas12a variants with expanded PAM profiles, many genomic loci, especially those with guanine‐cytosine (GC)‐rich PAMs, have remained inaccessible. This study develops a small RNA toxin‐aided strategy to evolve ErCas12a for targeting GC‐rich PAMs, resulting in the creation of enhanced ErCas12a (enErCas12a). EnErCas12a demonstrates the ability to recognize GC‐rich PAMs and target five times more PAM sequences than the wild‐type ErCas12a. Furthermore, enErCas12a achieves efficient gene editing in both bacterial and mammalian cells at various sites with non‐canonical PAMs, including GC‐rich PAMs such as GCCC, CGCC, and GGCC, which are inaccessible to previous Cas12a variants. Moreover, enErCas12a effectively targets PAM sequences with a GC content exceeding 75% in mammalian cells, providing a valuable alternative to the existing Cas12a toolkit. Importantly, enErCas12a maintains high specificity at targets with canonical PAMs, while also demonstrating enhanced specificity at targets with non‐canonical PAMs. Collectively, this work establishes enErCas12a as a promising tool for gene editing in both eukaryotes and prokaryotes.
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spelling doaj-art-eda50ec03a4b4b5cbd58008292a5043f2025-08-20T03:41:08ZengWileyAdvanced Science2198-38442025-08-011229n/an/a10.1002/advs.202417105Small RNA Toxin‐Assisted Evolution of GC‐Preferred ErCas12a for Enhanced Genome Targeting RangeZehua Chen0Junyuan Xue1Ziying Wang2Jinyuan Sun3Yinglu Cui4Tong Zhu5Huaiyi Yang6Ming Li7Bian Wu8AIM center College of Life Sciences and Technology Beijing University of Chemical Technology Institute of Microbiology Chinese Academy of Sciences Beijing 100101 ChinaState Key Laboratory of Microbial Diversity and Innovative Utilization Institute of Microbiology Chinese Academy of Sciences Beijing 100101 ChinaSenior Department of Orthopedics the Fourth Medical Center of PLA General Hospital Beijing 100000 ChinaUniversity of Chinese Academy of Sciences Beijing 100049 ChinaAIM center College of Life Sciences and Technology Beijing University of Chemical Technology Institute of Microbiology Chinese Academy of Sciences Beijing 100101 ChinaAIM center College of Life Sciences and Technology Beijing University of Chemical Technology Institute of Microbiology Chinese Academy of Sciences Beijing 100101 ChinaState Key Laboratory of Microbial Diversity and Innovative Utilization Institute of Microbiology Chinese Academy of Sciences Beijing 100101 ChinaState Key Laboratory of Microbial Diversity and Innovative Utilization Institute of Microbiology Chinese Academy of Sciences Beijing 100101 ChinaAIM center College of Life Sciences and Technology Beijing University of Chemical Technology Institute of Microbiology Chinese Academy of Sciences Beijing 100101 ChinaAbstract CRISPR/Cas12a, a promising gene editing technology, faces limitations due to its requirement for a thymine (T)‐rich protospacer adjacent motif (PAM). Despite the development of Cas12a variants with expanded PAM profiles, many genomic loci, especially those with guanine‐cytosine (GC)‐rich PAMs, have remained inaccessible. This study develops a small RNA toxin‐aided strategy to evolve ErCas12a for targeting GC‐rich PAMs, resulting in the creation of enhanced ErCas12a (enErCas12a). EnErCas12a demonstrates the ability to recognize GC‐rich PAMs and target five times more PAM sequences than the wild‐type ErCas12a. Furthermore, enErCas12a achieves efficient gene editing in both bacterial and mammalian cells at various sites with non‐canonical PAMs, including GC‐rich PAMs such as GCCC, CGCC, and GGCC, which are inaccessible to previous Cas12a variants. Moreover, enErCas12a effectively targets PAM sequences with a GC content exceeding 75% in mammalian cells, providing a valuable alternative to the existing Cas12a toolkit. Importantly, enErCas12a maintains high specificity at targets with canonical PAMs, while also demonstrating enhanced specificity at targets with non‐canonical PAMs. Collectively, this work establishes enErCas12a as a promising tool for gene editing in both eukaryotes and prokaryotes.https://doi.org/10.1002/advs.202417105CRISPR/Cas12aexpanded PAM profilesgene editingRNA toxin‐assisted evolution
spellingShingle Zehua Chen
Junyuan Xue
Ziying Wang
Jinyuan Sun
Yinglu Cui
Tong Zhu
Huaiyi Yang
Ming Li
Bian Wu
Small RNA Toxin‐Assisted Evolution of GC‐Preferred ErCas12a for Enhanced Genome Targeting Range
Advanced Science
CRISPR/Cas12a
expanded PAM profiles
gene editing
RNA toxin‐assisted evolution
title Small RNA Toxin‐Assisted Evolution of GC‐Preferred ErCas12a for Enhanced Genome Targeting Range
title_full Small RNA Toxin‐Assisted Evolution of GC‐Preferred ErCas12a for Enhanced Genome Targeting Range
title_fullStr Small RNA Toxin‐Assisted Evolution of GC‐Preferred ErCas12a for Enhanced Genome Targeting Range
title_full_unstemmed Small RNA Toxin‐Assisted Evolution of GC‐Preferred ErCas12a for Enhanced Genome Targeting Range
title_short Small RNA Toxin‐Assisted Evolution of GC‐Preferred ErCas12a for Enhanced Genome Targeting Range
title_sort small rna toxin assisted evolution of gc preferred ercas12a for enhanced genome targeting range
topic CRISPR/Cas12a
expanded PAM profiles
gene editing
RNA toxin‐assisted evolution
url https://doi.org/10.1002/advs.202417105
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