Evidence map›Paper›PMID 38280857›Full record

ArticleNature communications2024

Engineering a transposon-associated TnpB-ωRNA system for efficient gene editing and phenotypic correction of a tyrosinaemia mouse model.

Zhifang Li, Ruochen Guo, Xiaozhi Sun, Guoling Li, Zhuang Shao, Xiaona Huo, Rongrong Yang, Xinyu Liu, Xi Cao, Hainan Zhang and 11 more

Open access · goldAbstract read
In one paragraph

Article in Nature communications, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 40 papers.

0numbers the graph read from it
0cells of the map it votes in
40citing papers in PubMed
9.1field-weighted citation impact, top 1% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

40 citing papers in PubMed, 39 citations in OpenAlex.

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  20. Stepwise DNA unwinding gates TnpB genome-editing activity.bioRxiv : the preprint server for biology · 2026
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

21 authors at 8 institutions in 4 countries.

Zhifang Li *Lingang Laboratory, Shanghai, China.ORCID 0009-0000-7752-2454
Ruochen Guo *Lingang Laboratory, Shanghai, China.
Xiaozhi Sun *Lingang Laboratory, Shanghai, China.
Guoling Li *HuidaGene Therapeutics Inc, Shanghai, China.
Zhuang ShaoLingang Laboratory, Shanghai, China.
Xiaona HuoLingang Laboratory, Shanghai, China.
Rongrong YangLingang Laboratory, Shanghai, China.
Xinyu LiuInstitute of Neuroscience, State Key Laboratory of Neuroscience, Key Laboratory of Primate Neurobiology, CAS Center for Excellence in Brain Science and Intelligence Technology, Shanghai Institutes for Biological Sciences, Chinese Academy of Sciences, Shanghai, China.
Xi CaoLingang Laboratory, Shanghai, China.
Hainan ZhangHuidaGene Therapeutics Inc, Shanghai, China.
Weihong ZhangHuidaGene Therapeutics Inc, Shanghai, China.
Xiaoyin ZhangLingang Laboratory, Shanghai, China.
Shuangyu MaDepartment of Histoembryology, Genetics and Developmental Biology, Shanghai Key Laboratory of Reproductive Medicine, Shanghai JiaoTong University School of Medicine, Shanghai, China.
Meiling ZhangCenter for Reproductive Medicine, International Peace Maternity and Child Health Hospital, Innovative Research Team of High-level Local Universities in Shanghai, School of Medicine, Shanghai Jiao Tong University, Shanghai, China.
Yuanhua LiuInstitute of Neuroscience, State Key Laboratory of Neuroscience, Key Laboratory of Primate Neurobiology, CAS Center for Excellence in Brain Science and Intelligence Technology, Shanghai Institutes for Biological Sciences, Chinese Academy of Sciences, Shanghai, China.
Yinan YaoInstitute of Neuroscience, State Key Laboratory of Neuroscience, Key Laboratory of Primate Neurobiology, CAS Center for Excellence in Brain Science and Intelligence Technology, Shanghai Institutes for Biological Sciences, Chinese Academy of Sciences, Shanghai, China.
Jinqi ShiLingang Laboratory, Shanghai, China.
Hui YangInstitute of Neuroscience, State Key Laboratory of Neuroscience, Key Laboratory of Primate Neurobiology, CAS Center for Excellence in Brain Science and Intelligence Technology, Shanghai Institutes for Biological Sciences, Chinese Academy of Sciences, Shanghai, China.ORCID 0000-0002-8023-0230
Chunyi HuDepartment of Biological Sciences, National University of Singapore, Singapore, Singapore. hu_dbs@nus.edu.sg.ORCID 0000-0003-4509-4840
Yingsi ZhouHuidaGene Therapeutics Inc, Shanghai, China. yingsizhou@huidagene.com.ORCID 0000-0002-6541-2116
Chunlong XuLingang Laboratory, Shanghai, China. xucl@lglab.ac.cn.ORCID 0000-0003-3533-3465
Chinese Academy of Sciences · CNShanghai Center for Brain Science and Brain-Inspired Technology · CNShanghai Jiao Tong University · CNNational University of Singapore · SGNewGen Therapeutics (United States) · USNorthwest A&F University · CNShanghaiTech University · CNXLAB (Slovenia) · SI

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Transposon-associated ribonucleoprotein TnpB is known to be the ancestry endonuclease of diverse Cas12 effector proteins from type-V CRISPR system. Given its small size (408 aa), it is of interest to examine whether engineered TnpB could be used for efficient mammalian genome editing. Here, we showed that the gene editing activity of native TnpB from Deinococcus radiodurans (ISDra2 TnpB) in mouse embryos was already higher than previously identified small-sized Cas12f1. Further stepwise engineering of noncoding RNA (ωRNA or reRNA) component of TnpB significantly elevated the nuclease activity of TnpB. Notably, an optimized TnpB-ωRNA system could be efficiently delivered in vivo with single adeno-associated virus (AAV) and corrected the disease phenotype in a tyrosinaemia mouse model. Thus, the engineered miniature TnpB system represents a new addition to the current genome editing toolbox, with the unique feature of the smallest effector size that facilitate efficient AAV delivery for editing of cells and tissues.

Indexed as

Gene EditingTyrosinemiasAnimalsCRISPR-Cas SystemsMammalsMice

Identifiers

PMID38280857
PMCPMC10821889
OpenAlexW4391275707

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.