Evidence map›Paper›PMID 40643552›Full record

ArticleCells2025

Duchenne Muscular Dystrophy Patient iPSCs-Derived Skeletal Muscle Organoids Exhibit a Developmental Delay in Myogenic Progenitor Maturation.

Urs Kindler, Lampros Mavrommatis, Franziska Käppler, Dalya Gebrehiwet Hiluf, Stefanie Heilmann-Heimbach, Katrin Marcus, Thomas Günther Pomorski, Matthias Vorgerd, Beate Brand-Saberi, Holm Zaehres

Abstract read
In one paragraph

Article in Cells, 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. Organoids for disease modeling and treatment: state-of-the-art.Experimental hematology & oncology · 2026
    Review
  2. Article
  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

10 authors.

Urs KindlerDepartment of Anatomy and Molecular Embryology, Institute of Anatomy, Faculty of Medicine, Ruhr University Bochum, 44801 Bochum, NRW, Germany.ORCID 0000-0001-9676-6323
Lampros MavrommatisDepartment of Anatomy and Molecular Embryology, Institute of Anatomy, Faculty of Medicine, Ruhr University Bochum, 44801 Bochum, NRW, Germany.
Franziska KäpplerDepartment of Anatomy and Molecular Embryology, Institute of Anatomy, Faculty of Medicine, Ruhr University Bochum, 44801 Bochum, NRW, Germany.
Dalya Gebrehiwet HilufDepartment of Anatomy and Molecular Embryology, Institute of Anatomy, Faculty of Medicine, Ruhr University Bochum, 44801 Bochum, NRW, Germany.
Stefanie Heilmann-HeimbachInstitute of Human Genetics, NGS Core Facility, Faculty of Medicine, University of Bonn, 53127 Bonn, NRW, Germany.
Katrin MarcusMedical Proteom Center, Faculty of Medicine, Ruhr University Bochum, 44801 Bochum, NRW, Germany.ORCID 0000-0002-3313-8845
Thomas Günther PomorskiDepartment of Molecular Biochemistry, Faculty of Chemistry and Biochemistry, Ruhr University Bochum, 44801 Bochum, NRW, Germany.ORCID 0000-0002-4889-0829
Matthias VorgerdDepartment of Neurology with Heimer Institute for Muscle Research, Faculty of Medicine, University Hospital Bergmannsheil, Ruhr University Bochum, 44789 Bochum, NRW, Germany.ORCID 0000-0002-1782-4642
Beate Brand-SaberiDepartment of Anatomy and Molecular Embryology, Institute of Anatomy, Faculty of Medicine, Ruhr University Bochum, 44801 Bochum, NRW, Germany.ORCID 0000-0003-3309-9724
Holm ZaehresDepartment of Anatomy and Molecular Embryology, Institute of Anatomy, Faculty of Medicine, Ruhr University Bochum, 44801 Bochum, NRW, Germany.ORCID 0000-0001-8062-8428

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundDuchenne muscular dystrophy (DMD), which affects 1 in 3500 to 5000 newborn boys worldwide, is characterized by progressive skeletal muscle weakness and degeneration. The reduced muscle regeneration capacity presented by patients is associated with increased fibrosis. Satellite cells (SCs) are skeletal muscle stem cells that play an important role in adult muscle maintenance and regeneration. The absence or mutation of dystrophin in DMD is hypothesized to impair SC asymmetric division, leading to cell cycle arrest.

methodsTo overcome the limited availability of biopsies from DMD patients, we used our 3D skeletal muscle organoid (SMO) system, which delivers a stable population of myogenic progenitors (MPs) in dormant, activated, and committed stages, to perform SMO cultures using three DMD patient-derived iPSC lines.

resultsThe results of scRNA-seq analysis of three DMD SMO cultures versus two healthy, non-isogenic, SMO cultures indicate reduced MP populations with constant activation and differentiation, trending toward embryonic and immature myotubes. Mapping our data onto the human myogenic reference atlas, together with primary SC scRNA-seq data, indicated a more immature developmental stage of DMD organoid-derived MPs. DMD fibro-adipogenic progenitors (FAPs) appear to be activated in SMOs.

conclusionsOur organoid system provides a promising model for studying muscular dystrophies in vitro, especially in the case of early developmental onset, and a methodology for overcoming the bottleneck of limited patient material for skeletal muscle disease modeling.

Indexed as

Induced Pluripotent Stem CellsMuscle DevelopmentMuscle, SkeletalMuscular Dystrophy, DuchenneOrganoidsCell DifferentiationHumansMaleDuchenne muscular dystrophyFAPshuman-induced pluripotent stem cellsmyogenesismyogenic progenitorsorganoidssatellite cellsscRNA-seqskeletal muscle

Identifiers

PMID40643552
PMCPMC12249143

What Socratic 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 Socratic graph.