Guide RNA reprogramming facilitates minimized tracrRNA-dependent off-target and versatile CRISPR/Cas9 engineering

While innovative, current CRISPR-Cas9 systems face safety concerns and practical hurdles, notably sequence-independent, noncanonical off-targeting. We demonstrate that the crRNA:tracrRNA duplex in guide RNAs (gRNA) is both splittable and reprogrammable. This property, however, enables endogenous RNAs with crRNA-like sequences to hijack any gRNAs, causing low-frequency yet pervasive tracrRNA-dependent off-target (TDO) effects. Using machine learning trained on high-throughput gRNA variant screens, we derive optimal gRNA-designing rules and engineer crRNA variants mismatched to the human/mouse transcriptomes, thereby minimizing TDO. By leveraging splittability and reprogrammability, we develop reprogrammable tracrRNAs for CRISPRa-based mRNA detection and redesign scaffolds to curb PAM-less Cas9-mediated “self-editing”. We further create a separately expressed gRNA (segRNA) platform featuring split tracrRNAs and non-repetitive tandem crRNAs, enabling multiplexed editing of up to six genes and functional enhancer annotation in stem cells. Our findings uncover a previously overlooked off-target mechanism and offer versatile strategies to enhance the safety and utility of CRISPR systems. Leveraging the reprogrammability and splittability of CRISPR gRNAs, the authors find that endogenous crRNA-like RNAs hijack tracrRNAs to cause low-frequency yet pervasive tracrRNA-dependent off-target (TDO) effects. This same principle also offers versatile strategies to enhance CRISPR safety and utility.

Authors

Institutions

Publication Details

Journal
Nature Communications
Published
2026-06-17
DOI
https://doi.org/10.1038/s41467-026-74520-z
Primary Topic
CRISPR and Genetic Engineering
Type
article
Field-Weighted Citation Impact
0.00
Controls
|||
ALL TIME
JAN
FEB
MAR
APR
MAY
JUN
JUL
AUG
SEP
OCT
article

Guide RNA reprogramming facilitates minimized tracrRNA-dependent off-target and versatile CRISPR/Cas9 engineering

Pengfei Lan, Qian Bian, Peixiang Ma, Sicong Huang et al.
Nature Communications
CRISPR and Genetic Engineering
article

Guide RNA reprogramming facilitates minimized tracrRNA-dependent off-target and versatile CRISPR/Cas9 engineering

Pengfei Lan, Qian Bian, Peixiang Ma, Sicong Huang, Xingxu Huang, J Chen, Ligang Wu, Yunbo Qiao, Tian‐Lin Cheng, Wenxia Yu, Xiangping Dai, Jianxiang Lin, J Z Guo, G Greg Wang, Xiaoguo Zheng, Fang Yu, Jiao Wei, Xinyi Tan, Yu Zhang
article en

Abstract

While innovative, current CRISPR-Cas9 systems face safety concerns and practical hurdles, notably sequence-independent, noncanonical off-targeting. We demonstrate that the crRNA:tracrRNA duplex in guide RNAs (gRNA) is both splittable and reprogrammable. This property, however, enables endogenous RNAs with crRNA-like sequences to hijack any gRNAs, causing low-frequency yet pervasive tracrRNA-dependent off-target (TDO) effects. Using machine learning trained on high-throughput gRNA variant screens, we derive optimal gRNA-designing rules and engineer crRNA variants mismatched to the human/mouse transcriptomes, thereby minimizing TDO. By leveraging splittability and reprogrammability, we develop reprogrammable tracrRNAs for CRISPRa-based mRNA detection and redesign scaffolds to curb PAM-less Cas9-mediated “self-editing”. We further create a separately expressed gRNA (segRNA) platform featuring split tracrRNAs and non-repetitive tandem crRNAs, enabling multiplexed editing of up to six genes and functional enhancer annotation in stem cells. Our findings uncover a previously overlooked off-target mechanism and offer versatile strategies to enhance the safety and utility of CRISPR systems. Leveraging the reprogrammability and splittability of CRISPR gRNAs, the authors find that endogenous crRNA-like RNAs hijack tracrRNAs to cause low-frequency yet pervasive tracrRNA-dependent off-target (TDO) effects. This same principle also offers versatile strategies to enhance CRISPR safety and utility.

Nature Communications
Renji Hospital (CN), ShanghaiTech University (CN), Shanghai Ninth People's Hospital (CN), International Peace Maternity & Child Health Hospital (CN), Center for Excellence in Molecular Cell Science (CN), Changzhou No.2 People's Hospital (CN), Children's Hospital of Fudan University (CN), Qilu Hospital of Shandong University (CN), University of Chinese Academy of Sciences (CN), First Affiliated Hospital Zhejiang University (CN), Zhejiang University (CN)
Openalex Percentile: Top 11%
CRISPR and Genetic Engineering
AI Navigator

Ask Laika to Summarize, Analyze, and Connect papers live on the map.

Summarize Papers & Methodologies

Extract key findings, datasets, and comparative methods across publications.

Benchmark Rankings & Visual Analytics

Rank top research institutions, authors, funders, topics, and journals by Field-Weighted Citation Impact (FWCI) and paper volume with instant charts.

Connect Distant Disciplines

Bridge topological clusters on the map to find hidden collaborative intersections.