Highly parallel profiling of the activities and specificities of Cas12a variants in human cells

Abstract Several Cas12a variants have been developed to broaden its targeting range, improve the gene editing specificity or the efficiency. However, selecting the appropriate Cas12a among the many orthologs for a given target sequence remains difficult. Here, we perform high-throughput analyses to...

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Main Authors: Peng Chen, Yankang Wu, Hongjian Wang, Huan Liu, Jin Zhou, Jingli Chen, Jun Lei, Zaiqiao Sun, Chonil Paek, Lei Yin
Format: Article
Language:English
Published: Nature Portfolio 2025-03-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-025-57150-9
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author Peng Chen
Yankang Wu
Hongjian Wang
Huan Liu
Jin Zhou
Jingli Chen
Jun Lei
Zaiqiao Sun
Chonil Paek
Lei Yin
author_facet Peng Chen
Yankang Wu
Hongjian Wang
Huan Liu
Jin Zhou
Jingli Chen
Jun Lei
Zaiqiao Sun
Chonil Paek
Lei Yin
author_sort Peng Chen
collection DOAJ
description Abstract Several Cas12a variants have been developed to broaden its targeting range, improve the gene editing specificity or the efficiency. However, selecting the appropriate Cas12a among the many orthologs for a given target sequence remains difficult. Here, we perform high-throughput analyses to evaluate the activity and compatibility with specific PAMs of 24 Cas12a variants and develop deep learning models for these Cas12a variants to predict gene editing activities at target sequences of interest. Furthermore, we reveal and enhance the truncation in the integrated tag sequence that may hinder off-targeting detection for Cas12a by GUIDE-seq. This enhanced system, which we term enGUIDE-seq, is used to evaluate and compare the off-targeting and translocations of these Cas12a variants.
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institution Kabale University
issn 2041-1723
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publishDate 2025-03-01
publisher Nature Portfolio
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series Nature Communications
spelling doaj-art-de1fcf27aac84a67b0606cce6e7ceae82025-08-20T03:40:50ZengNature PortfolioNature Communications2041-17232025-03-0116111310.1038/s41467-025-57150-9Highly parallel profiling of the activities and specificities of Cas12a variants in human cellsPeng Chen0Yankang Wu1Hongjian Wang2Huan Liu3Jin Zhou4Jingli Chen5Jun Lei6Zaiqiao Sun7Chonil Paek8Lei Yin9Department of Pediatric Research Institute; Ministry of Education Key Laboratory of Child Development and Disorders, National Clinical Research Center for Child Health and Disorders, China International Science and Technology Cooperation Base of Child Development and Critical Disorders, Children’s Hospital of Chongqing Medical University, School of Basic Medical Sciences, Chongqing Medical UniversityState Key Laboratory of Virology, Hubei Key Laboratory of Cell Homeostasis, College of Life Sciences, Wuhan UniversityState Key Laboratory of Virology, Hubei Key Laboratory of Cell Homeostasis, College of Life Sciences, Wuhan UniversityState Key Laboratory of Virology, Hubei Key Laboratory of Cell Homeostasis, College of Life Sciences, Wuhan UniversityDepartment of Pediatric Research Institute; Ministry of Education Key Laboratory of Child Development and Disorders, National Clinical Research Center for Child Health and Disorders, China International Science and Technology Cooperation Base of Child Development and Critical Disorders, Children’s Hospital of Chongqing Medical University, School of Basic Medical Sciences, Chongqing Medical UniversitySchool of Medicine, Wuhan University of Science and TechnologyDepartment of Pediatric Research Institute; Ministry of Education Key Laboratory of Child Development and Disorders, National Clinical Research Center for Child Health and Disorders, China International Science and Technology Cooperation Base of Child Development and Critical Disorders, Children’s Hospital of Chongqing Medical University, School of Basic Medical Sciences, Chongqing Medical UniversityState Key Laboratory of Virology, Hubei Key Laboratory of Cell Homeostasis, College of Life Sciences, Wuhan UniversityState Key Laboratory of Virology, Hubei Key Laboratory of Cell Homeostasis, College of Life Sciences, Wuhan UniversityDepartment of Pediatric Research Institute; Ministry of Education Key Laboratory of Child Development and Disorders, National Clinical Research Center for Child Health and Disorders, China International Science and Technology Cooperation Base of Child Development and Critical Disorders, Children’s Hospital of Chongqing Medical University, School of Basic Medical Sciences, Chongqing Medical UniversityAbstract Several Cas12a variants have been developed to broaden its targeting range, improve the gene editing specificity or the efficiency. However, selecting the appropriate Cas12a among the many orthologs for a given target sequence remains difficult. Here, we perform high-throughput analyses to evaluate the activity and compatibility with specific PAMs of 24 Cas12a variants and develop deep learning models for these Cas12a variants to predict gene editing activities at target sequences of interest. Furthermore, we reveal and enhance the truncation in the integrated tag sequence that may hinder off-targeting detection for Cas12a by GUIDE-seq. This enhanced system, which we term enGUIDE-seq, is used to evaluate and compare the off-targeting and translocations of these Cas12a variants.https://doi.org/10.1038/s41467-025-57150-9
spellingShingle Peng Chen
Yankang Wu
Hongjian Wang
Huan Liu
Jin Zhou
Jingli Chen
Jun Lei
Zaiqiao Sun
Chonil Paek
Lei Yin
Highly parallel profiling of the activities and specificities of Cas12a variants in human cells
Nature Communications
title Highly parallel profiling of the activities and specificities of Cas12a variants in human cells
title_full Highly parallel profiling of the activities and specificities of Cas12a variants in human cells
title_fullStr Highly parallel profiling of the activities and specificities of Cas12a variants in human cells
title_full_unstemmed Highly parallel profiling of the activities and specificities of Cas12a variants in human cells
title_short Highly parallel profiling of the activities and specificities of Cas12a variants in human cells
title_sort highly parallel profiling of the activities and specificities of cas12a variants in human cells
url https://doi.org/10.1038/s41467-025-57150-9
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