Evidence map›Paper›PMID 40970088›Full record

ReviewFrontiers in cell and developmental biology2025

Stem/progenitor cell-based therapy for Duchenne muscular dystrophy.

Tsukasa Tominari, Chaitra Sathyaprakash, Yoshitsugu Aoki

Abstract readReview
In one paragraph

Review in Frontiers in cell and developmental biology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed.

  1. 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

3 authors.

Tsukasa TominariDepartment of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Tokyo, Japan.
Chaitra SathyaprakashDepartment of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Tokyo, Japan.
Yoshitsugu AokiDepartment of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Tokyo, Japan.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Duchenne muscular dystrophy is a genetic disease where loss of sarcolemma-associated protein, dystrophin, leads to progressive muscle wasting, and eventual loss of life from complications linked to cardiac deficits. Currently, numerous molecular therapies to restore dystrophin have entered clinical trials. However, the therapeutic benefits of these strategies in promoting tissue regeneration and reducing fibrosis remain limited. Stem/progenitor cell-based therapy in DMD patients is a promising strategy to promote muscle regeneration, though the conditions of transplantation and pre-treatments of numerous cell types are still being optimized. Several cell types with different properties and origins, such as myogenic stem/progenitor cells, mesenchymal stem cells (MSCs), and induced pluripotent stem cells (iPSCs), have been studied for treating DMD. Myogenic stem/progenitor cells derived from healthy donors are expected to restore the number of myofibers as well as dystrophin expression in DMD muscles. MSCs derived from various tissues, including umbilical cord, have immunosuppressive properties and are expected to ameliorate DMD phenotypes in combination with other gene therapies. In this review, we will summarize the challenges that must be overcome to allow for successful DMD muscle tissue regeneration and review the latest findings in stem/progenitor cell-based DMD therapy. We will focus on the pre-conditioning of cells for replacement therapies and treatment of the disease niche to improve muscle fiber integration.

Indexed as

cardiac muscleduchenne muscular dystrophy (DMD)dystrophinskeletal musclestem cell therapies

Identifiers

PMID40970088
PMCPMC12441038

What OpenQuestion holds

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Registered trials

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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.