Evidence map›Paper›PMID 40056909›Full record

ArticleCell reports. Medicine2025

Engineered human myogenic cells in hydrogels generate innervated vascularized myofibers within dystrophic mouse muscle on long-term engraftment.

Anna Kowala, James Boot, Jinhong Meng, Charles A Mein, Olivier Pourquié, John T Connelly, Jennifer E Morgan, Yung-Yao Lin

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. Review
  2. Review
  3. Stem/progenitor cell-based therapy for Duchenne muscular dystrophy.Frontiers in cell and developmental biology · 2025
    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

8 authors.

Anna KowalaCentre for Genomics and Child Health, Blizard Institute, Faculty of Medicine and Dentistry, Queen Mary University of London, 4 Newark Street, London E1 2AT, UK; Stem Cell Laboratory, National Bowel Research Centre, Blizard Institute, Faculty of Medicine and Dentistry, Queen Mary University of London, 2 Newark Street, London E1 2AT, UK; Centre for Predictive in vitro Models, Queen Mary University of London, Mile End Road, London E1 4NS, UK; UCL Great Ormond Street Institute of Child Health, 30 Guilford Street, London WC1N 1EH, UK.
James BootBarts and the London Genome Centre, Faculty of Medicine and Dentistry, Blizard Institute, London, UK.
Jinhong MengUCL Great Ormond Street Institute of Child Health, 30 Guilford Street, London WC1N 1EH, UK; NIHR Biomedical Research Centre at Great Ormond Street Hospital, Great Ormond Street, London, UK.
Charles A MeinBarts and the London Genome Centre, Faculty of Medicine and Dentistry, Blizard Institute, London, UK.
Olivier PourquiéDepartment of Genetics, Harvard Medical School and Department of Pathology, Brigham and Women's Hospital, 60 Fenwood Road, Boston, MA, USA.
John T ConnellyCentre for Predictive in vitro Models, Queen Mary University of London, Mile End Road, London E1 4NS, UK; Centre for Cell Biology and Cutaneous Research, Blizard Institute, Faculty of Medicine and Dentistry, Queen Mary University of London, 4 Newark Street, London E1 2AT, UK.
Jennifer E MorganUCL Great Ormond Street Institute of Child Health, 30 Guilford Street, London WC1N 1EH, UK; NIHR Biomedical Research Centre at Great Ormond Street Hospital, Great Ormond Street, London, UK.
Yung-Yao LinCentre for Genomics and Child Health, Blizard Institute, Faculty of Medicine and Dentistry, Queen Mary University of London, 4 Newark Street, London E1 2AT, UK; Stem Cell Laboratory, National Bowel Research Centre, Blizard Institute, Faculty of Medicine and Dentistry, Queen Mary University of London, 2 Newark Street, London E1 2AT, UK; Centre for Predictive in vitro Models, Queen Mary University of London, Mile End Road, London E1 4NS, UK. Electronic address: yy.lin@qmul.ac.uk.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Transplantation of human myogenic progenitor cells (MPCs) is a promising therapeutic strategy for treating muscle-wasting diseases, e.g., Duchenne muscular dystrophy (DMD). To increase engraftment efficiency of donor stem cells, modulation of host muscles is required, significantly limiting their clinical translation. Here, we develop a clinically relevant transplantation strategy synergizing hydrogel-mediated delivery and engineered human MPCs generated from CRISPR-corrected DMD patient-derived pluripotent stem cells. We demonstrate that donor-derived human myofibers produce full-length dystrophin at 4 weeks and 5-6 months (long-term) after transplantation in the unmodulated muscles of the dystrophin-deficient mouse model of DMD. Remarkably, human myofibers are innervated by mouse motor neurons forming neuromuscular junctions and supported by vascularization after long-term engraftment in dystrophic mice. PAX7+ cells of human origin populate the satellite cell niche. There was no evidence of tumorigenesis in mice engrafted with hydrogel-encapsulated human MPCs. Our results provide a proof of concept in developing hydrogel-based cell therapy for muscle-wasting diseases.

Indexed as

Cell EngineeringHydrogelsMuscle Fibers, SkeletalMuscular Dystrophy, DuchenneAnimalsDisease Models, AnimalDystrophinHumansMiceMice, Inbred mdxMuscle DevelopmentMuscle, SkeletalDystrophinHydrogelsbiomaterialsCRISPRDuchenne muscular dystrophydystrophinhydrogelsinnervationregenerative medicinestem cellsvascularizationxenoengraftment

Identifiers

PMID40056909
PMCPMC11970389

What OpenQuestion holds

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LicenceCC BY
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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.