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
- Pengfei Lan (ORCID: https://orcid.org/0000-0002-7654-2717)
- Qian Bian (ORCID: https://orcid.org/0000-0001-7022-2213)
- Peixiang Ma (ORCID: https://orcid.org/0000-0001-6794-1663)
- Sicong Huang (ORCID: https://orcid.org/0000-0003-0854-4734)
- Xingxu Huang (ORCID: https://orcid.org/0000-0001-8934-1247)
- J Chen
- Ligang Wu (ORCID: https://orcid.org/0000-0003-4010-9118)
- Yunbo Qiao (ORCID: https://orcid.org/0000-0002-8391-5868)
- Tian‐Lin Cheng (ORCID: https://orcid.org/0000-0003-0680-6710)
- Wenxia Yu
- Xiangping Dai
- Jianxiang Lin
- J Z Guo
- G Greg Wang
- Xiaoguo Zheng (ORCID: https://orcid.org/0009-0003-3181-7398)
- Fang Yu
- Jiao Wei
- Xinyi Tan
- Yu Zhang
Institutions
- 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)
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