Evidence map›Paper›PMID 40147446›Full record

ArticleCell reports. Medicine2025

Single-cut gene therapy in a one-step generated rhesus monkey model of Duchenne muscular dystrophy.

Raoxian Bai, Wenting Guo, Ting Zhang, Shuaiwei Ren, Jie Liu, Puhao Xiao, Junyu Zhang, Wenjie Sun, Jiao Yang, Yue Ma and 8 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Cell reports. Medicine, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing 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

3 citing papers in PubMed.

  1. Article
  2. Article
  3. Article
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

18 authors.

Raoxian BaiState Key Laboratory of Primate Biomedical Research, Institute of Primate Translational Medicine, Kunming University of Science and Technology, Kunming 650500, China; Faculty of Life Science and Technology, Kunming University of Science and Technology, Kunming 650500, China; Yunnan Key Laboratory of Primate Biomedical Research, Kunming 650500, China.
Wenting GuoState Key Laboratory of Primate Biomedical Research, Institute of Primate Translational Medicine, Kunming University of Science and Technology, Kunming 650500, China; Yunnan Key Laboratory of Primate Biomedical Research, Kunming 650500, China.
Ting ZhangState Key Laboratory of Primate Biomedical Research, Institute of Primate Translational Medicine, Kunming University of Science and Technology, Kunming 650500, China; Yunnan Key Laboratory of Primate Biomedical Research, Kunming 650500, China.
Shuaiwei RenState Key Laboratory of Primate Biomedical Research, Institute of Primate Translational Medicine, Kunming University of Science and Technology, Kunming 650500, China.
Jie LiuState Key Laboratory of Primate Biomedical Research, Institute of Primate Translational Medicine, Kunming University of Science and Technology, Kunming 650500, China; Yunnan Key Laboratory of Primate Biomedical Research, Kunming 650500, China.
Puhao XiaoState Key Laboratory of Primate Biomedical Research, Institute of Primate Translational Medicine, Kunming University of Science and Technology, Kunming 650500, China; Southwest United Graduate School, Kunming 650092, China.
Junyu ZhangState Key Laboratory of Primate Biomedical Research, Institute of Primate Translational Medicine, Kunming University of Science and Technology, Kunming 650500, China; Southwest United Graduate School, Kunming 650092, China.
Wenjie SunState Key Laboratory of Primate Biomedical Research, Institute of Primate Translational Medicine, Kunming University of Science and Technology, Kunming 650500, China; Yunnan Key Laboratory of Primate Biomedical Research, Kunming 650500, China.
Jiao YangState Key Laboratory of Primate Biomedical Research, Institute of Primate Translational Medicine, Kunming University of Science and Technology, Kunming 650500, China.
Yue MaState Key Laboratory of Primate Biomedical Research, Institute of Primate Translational Medicine, Kunming University of Science and Technology, Kunming 650500, China.
Siyu LiuState Key Laboratory of Primate Biomedical Research, Institute of Primate Translational Medicine, Kunming University of Science and Technology, Kunming 650500, China.
Chaoran ZhouState Key Laboratory of Primate Biomedical Research, Institute of Primate Translational Medicine, Kunming University of Science and Technology, Kunming 650500, China; Southwest United Graduate School, Kunming 650092, China.
Shangang LiState Key Laboratory of Primate Biomedical Research, Institute of Primate Translational Medicine, Kunming University of Science and Technology, Kunming 650500, China; Yunnan Key Laboratory of Primate Biomedical Research, Kunming 650500, China.
Hong WangState Key Laboratory of Primate Biomedical Research, Institute of Primate Translational Medicine, Kunming University of Science and Technology, Kunming 650500, China; Yunnan Key Laboratory of Primate Biomedical Research, Kunming 650500, China.
Shu ZhangDepartment of Neurology, First Medical Center of Chinese PLA General Hospital, Beijing 100853, China.
Weizhi JiState Key Laboratory of Primate Biomedical Research, Institute of Primate Translational Medicine, Kunming University of Science and Technology, Kunming 650500, China; Yunnan Key Laboratory of Primate Biomedical Research, Kunming 650500, China. Electronic address: wji@lpbr.cn.
Shiwen WuDepartment of Neurology, First Medical Center of Chinese PLA General Hospital, Beijing 100853, China. Electronic address: wu_shiwen@outlook.com.
Yongchang ChenState Key Laboratory of Primate Biomedical Research, Institute of Primate Translational Medicine, Kunming University of Science and Technology, Kunming 650500, China; Faculty of Life Science and Technology, Kunming University of Science and Technology, Kunming 650500, China; Yunnan Key Laboratory of Primate Biomedical Research, Kunming 650500, China; Southwest United Graduate School, Kunming 650092, China. Electronic address: chenyc@lpbr.cn.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Progress in Duchenne muscular dystrophy (DMD) treatment is hindered by the lack of animal models that closely replicate human pathology and enable the evaluation of therapy efficacy and safety based on these models. To address this need, we optimize the generation of nonhuman primate DMD models, reducing the development time from 6 to 7 years to under 1 year, enabling the rapid generation of DMD monkey models. These models closely mimic human DMD pathology and motor dysfunction, making them suitable for testing gene therapies. Using these models, we develop a single-cut gene therapy strategy that can be directly applied to humans. This treatment restores dystrophin expression, improves pathological features, and enhances motor abilities in DMD monkeys, with effects lasting at least 1.5 years. In conclusion, we achieve the rapid generation of DMD monkey models and demonstrate that our gene therapy approach is effective and holds significant potential for clinical application.

Indexed as

Genetic TherapyMuscular Dystrophy, DuchenneAnimalsDisease Models, AnimalDystrophinHumansMacaca mulattaMaleMuscle, SkeletalDystrophinCas12i(Max)Duchenne muscular dystrophydystrophin restorationexon reframingexon skippinggene correctiongene therapynonhuman primate disease modelsingle-cutsystemic gene delivery

Identifiers

PMID40147446
PMCPMC12047476

What Socratic holds

Textmetadata
LicenceCC BY-NC-ND
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 Socratic graph.