Evidence map›Paper›PMID 42432197›Full record

ArticleNature biotechnology2026

Efficient in vivo cytosine base editing using virus-like particles with uracil DNA glycosylase inhibition.

Junjie Zhu, Lin Ding, Kai-Ming Liu, Xingyu Zhu, Jifang Li, Buer Ha, Bao-Qing Gao, Xujiao Zhou, Chengfang Liu, Jia Wei and 10 more

Abstract read
PubMed Publisher
In one paragraph

Article in Nature biotechnology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
–field-weighted citation impact
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

2 citing papers in PubMed.

  1. Article
  2. Advancements in CRISPR-basedFrontiers in genome editing · 2026
    Review
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

20 authors.

Junjie Zhu *Gene Editing Center, School of Life Science and Technology, ShanghaiTech University, Shanghai, China.
Lin Ding *Gene Editing Center, School of Life Science and Technology, ShanghaiTech University, Shanghai, China.
Kai-Ming Liu *Center for Molecular Medicine, Children's Hospital of Fudan University and Shanghai Key Laboratory of Medical Epigenetics, International Laboratory of Medical Epigenetics and Metabolism, Ministry of Science and Technology, Institutes of Biomedical Sciences, Fudan University, Shanghai, China.
Xingyu Zhu *Department of Ophthalmology, Eye and ENT Hospital, State Key Laboratory of Brain Function and Disorders, MOE Frontiers Center for Brain Science, Fudan University, Shanghai, China.
Jifang Li *Gene Editing Center, School of Life Science and Technology, ShanghaiTech University, Shanghai, China.
Buer HaGene Editing Center, School of Life Science and Technology, ShanghaiTech University, Shanghai, China.
Bao-Qing GaoCenter for Molecular Medicine, Children's Hospital of Fudan University and Shanghai Key Laboratory of Medical Epigenetics, International Laboratory of Medical Epigenetics and Metabolism, Ministry of Science and Technology, Institutes of Biomedical Sciences, Fudan University, Shanghai, China.
Xujiao ZhouDepartment of Ophthalmology, Eye and ENT Hospital, State Key Laboratory of Brain Function and Disorders, MOE Frontiers Center for Brain Science, Fudan University, Shanghai, China.
Chengfang LiuGene Editing Center, School of Life Science and Technology, ShanghaiTech University, Shanghai, China.
Jia WeiCenter for Molecular Medicine, Children's Hospital of Fudan University and Shanghai Key Laboratory of Medical Epigenetics, International Laboratory of Medical Epigenetics and Metabolism, Ministry of Science and Technology, Institutes of Biomedical Sciences, Fudan University, Shanghai, China.
Shuangyu MaDepartment of Ophthalmology, Eye and ENT Hospital, State Key Laboratory of Brain Function and Disorders, MOE Frontiers Center for Brain Science, Fudan University, Shanghai, China.
Wenchao XuGene Editing Center, School of Life Science and Technology, ShanghaiTech University, Shanghai, China.
Lingyun KongDepartment of Ophthalmology, Eye and ENT Hospital, State Key Laboratory of Brain Function and Disorders, MOE Frontiers Center for Brain Science, Fudan University, Shanghai, China.
Jing WuGene Editing Center, School of Life Science and Technology, ShanghaiTech University, Shanghai, China.
Xiaodun MouCorrectSequence Therapeutics, Shanghai, China.ORCID http://orcid.org/0009-0007-5449-712X
Bei YangGene Editing Center, School of Life Science and Technology, ShanghaiTech University, Shanghai, China.ORCID http://orcid.org/0000-0001-5389-3859
Lijie WangCorrectSequence Therapeutics, Shanghai, China.
Jiaxu HongDepartment of Ophthalmology, Eye and ENT Hospital, State Key Laboratory of Brain Function and Disorders, MOE Frontiers Center for Brain Science, Fudan University, Shanghai, China. jiaxu.hong@fdeent.org.ORCID http://orcid.org/0000-0001-9912-633X
Li YangCenter for Molecular Medicine, Children's Hospital of Fudan University and Shanghai Key Laboratory of Medical Epigenetics, International Laboratory of Medical Epigenetics and Metabolism, Ministry of Science and Technology, Institutes of Biomedical Sciences, Fudan University, Shanghai, China. liyang_fudan@fudan.edu.cn.ORCID http://orcid.org/0000-0001-8833-7473
Jia ChenGene Editing Center, School of Life Science and Technology, ShanghaiTech University, Shanghai, China. chenjia@shanghaitech.edu.cn.ORCID http://orcid.org/0000-0002-4242-2899

Funding

National Natural Science Foundation of China (National Science Foundation of China) 32430018National Natural Science Foundation of China (National Science Foundation of China) 82171102
6 · The paper itself

Abstract

Virus-like particles (VLPs) are promising for delivering genome editors, yet the in vivo in vivo efficacy of VLP-mediated cytosine base editing remains limited. Here we identified insufficient inhibition of uracil DNA glycosylases as the underlying mechanism of low cytosine base editor (CBE) editing efficiencies in vivo. We engineered a previously reported CBE, transformer base editor (tBE), and developed a VLP delivery system to enhance the recruitment of uracil DNA glycosylase inhibitor proteins. tBE-VLPs achieved robust C-to-T editing in mouse liver and retina. A single injection achieved, on average, 46.0% editing at mPcsk9 and 64.2% at mHpd in the liver, as well as 24.2% at mVegfa in the retinal pigment epithelium, resulting in marked therapeutic benefits in mouse disease models. tBE-VLP4 induced no detectable off-target edits in vitro or in vivo and demonstrated superior specificity compared to AAV or lipid nanoparticle mRNA delivery. Our work establishes tBE-VLP4 as a precise, efficient system for in vivo cytosine base editing.

Identifiers

PMID42432197

What OpenQuestion holds

Textmetadata
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.