Evidence map›Paper›PMID 42277318›Full record

ArticleNature biomedical engineering2026

Skeletal-muscle-targeted non-viral delivery of full-length DMD mRNA for Duchenne muscular dystrophy.

Yu Tian, Yutong Liu, Yuhao Tong, Kristin Huntoon, Liqun Yang, Junfeng Shi, Yifan Ma, Shiyan Dong, Seong Dong Jeong, Andreanne Poppy Estania and 22 more

Abstract read
PubMed Publisher
In one paragraph

Article in Nature biomedical engineering, 2026. 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. Review
  2. AI meets high-throughput screening of LNPs.Nature biomedical engineering · 2026
    Article
  3. Engineering stable, off-the-shelf cellular depots.Nature biomedical engineering · 2026
    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

32 authors.

Yu Tian *School of Chemical Biology and Biotechnology, Peking University Shenzhen Graduate School, Shenzhen, China.ORCID http://orcid.org/0009-0004-1919-0614
Yutong Liu *School of Chemical Biology and Biotechnology, Peking University Shenzhen Graduate School, Shenzhen, China.
Yuhao Tong *School of Chemical Biology and Biotechnology, Peking University Shenzhen Graduate School, Shenzhen, China.
Kristin Huntoon *Department of Neurosurgery, University of Arizona, Tucson, AZ, USA.
Liqun YangSchool of Chemical Biology and Biotechnology, Peking University Shenzhen Graduate School, Shenzhen, China.ORCID http://orcid.org/0009-0007-6564-501X
Junfeng ShiSpot Biosystems, Palo Alto, CA, USA.
Yifan MaDepartment of Radiation Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.ORCID http://orcid.org/0000-0002-9371-4365
Shiyan DongDepartment of Radiation Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.ORCID http://orcid.org/0000-0003-2912-8895
Seong Dong JeongDepartment of Neurosurgery, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.ORCID http://orcid.org/0009-0003-5163-3924
Andreanne Poppy EstaniaSchool of Chemical Biology and Biotechnology, Peking University Shenzhen Graduate School, Shenzhen, China.
You YiSchool of Chemical Biology and Biotechnology, Peking University Shenzhen Graduate School, Shenzhen, China.
Kwang Joo KwakSpot Biosystems, Palo Alto, CA, USA.
Yifan WangDepartment of Radiation Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.ORCID http://orcid.org/0000-0001-7387-0654
Annette WuDepartment of Radiation Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.ORCID http://orcid.org/0009-0005-8343-4748
Jared L EdwardsDepartment of Radiation Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.ORCID http://orcid.org/0009-0008-7604-3441
Xiaotian WangDepartment of Radiation Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
Yen-Tzu ChangDepartment of Radiation Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
Jianhong CaoSchool of Chemical Biology and Biotechnology, Peking University Shenzhen Graduate School, Shenzhen, China.
Huan ZhangSchool of Life Sciences, Jilin University, Changchun, China.
Man SunSchool of Life Sciences, Jilin University, Changchun, China.
Adam J GrippinDepartment of Radiation Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.ORCID http://orcid.org/0000-0002-8076-4240
Zhaogang YangSchool of Life Sciences, Jilin University, Changchun, China.ORCID http://orcid.org/0000-0002-6350-4455
Wen-Jing LuShenzhen Key Laboratory of Cardiovascular Disease, Fuwai Hospital, Chinese Academy of Medical Sciences, Shenzhen, China.
Ly James LeeSpot Biosystems, Palo Alto, CA, USA.ORCID http://orcid.org/0000-0002-3589-0280
Eman BahraniDepartments of Dermatology and Pathology, Vanderbilt University Medical Center, Nashville, TN, USA.
Hongjia ZhangBeijing Laboratory for Cardiovascular Precision Medicine, The Key Laboratory of Biomedical Engineering for Cardiovascular Disease Research, Ministry of Education, Beijing Anzhen Hospital, Capital Medical University, Beijing, China.
Long BaiInstitute of Translational Medicine, Shanghai University, Shanghai, China.
Patricia K NguyenDepartment of Medicine, Division of Cardiovascular Medicine, Stanford University School of Medicine, Stanford, CA, USA.
Wen JiangDepartment of Radiation Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA. wjiang4@mdanderson.org.ORCID http://orcid.org/0000-0001-9154-633X
Feng LanShenzhen Key Laboratory of Cardiovascular Disease, Fuwai Hospital, Chinese Academy of Medical Sciences, Shenzhen, China. Fenglan@fuwai.com.ORCID http://orcid.org/0000-0002-9038-4014
Betty Y S KimDepartment of Neurosurgery, The University of Texas MD Anderson Cancer Center, Houston, TX, USA. bykim@mdanderson.org.ORCID http://orcid.org/0000-0001-6890-8355
Andrew S LeeSchool of Chemical Biology and Biotechnology, Peking University Shenzhen Graduate School, Shenzhen, China. Alee@pku.edu.cn.ORCID http://orcid.org/0000-0001-9169-1481

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Duchenne muscular dystrophy (DMD) is a severe, progressive muscle-wasting disorder caused by mutations in the DMD gene, which encodes dystrophin. Although gene therapy using viral vectors has shown promise for the treatment of DMD, the clinical application of viral gene therapies is limited by vector toxicity, immunogenicity and the inability to package full-length dystrophin. Recent advances in messenger RNA (mRNA) technology offer a non-integrating, transient approach to restoring protein expression. Here we report the systemic delivery of skeletal-muscle-targeted full-length DMD mRNA in a murine model of DMD using allogenically engineered targeting extracellular vesicles (DMD t-EVs). This approach restores the endogenous translation of wild-type dystrophin and substantially improves muscle function. We further demonstrate the safety and biocompatibility of DMD t-EVs in non-human primates, supporting their translational potential. These findings highlight the promise of mRNA-loaded extracellular vesicles as a therapeutic platform for treating genetic disorders involving large, difficult-to-package genes.

Identifiers

PMID42277318

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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.