[iPS cell-based therapy for muscular disorders].
Sakurai H.
Animal Study, published in Rinsho Shinkeigaku (2025) — summary generated from the PubMed abstract.
Evidence from laboratory and animal studies provides groundwork for understanding mechanisms and potential before human studies continue.
- Level A · Stronger Clinical Evidence
- Level B · Emerging clinical evidence with positive signals
- Level C · Early human research exploring benefits
- Level D · Scientific groundwork from lab and animal studies
- Emerging · Emerging topic under active research
This page is generated from the PubMed record. The Thai description is an automated summary of bibliographic fields and the abstract, not a full translation, and is not medical advice.
- Study type
- Animal Study
- Journal
- Rinsho Shinkeigaku (2025)
- Country
- Japan
- Reported sample size
- —
- Source database
- PubMed
- PMID
- 41016756
- DOI
- 10.5692/clinicalneurol.cn-002114
Abstract (original English)
Induced pluripotent stem cells (iPSCs) have been used in research for the development of treatments for various intractable diseases due to their unlimited proliferative and multipotent potential. We are aiming to develop novel therapies for intractable muscular diseases using iPS cells by two approaches i.e. cell therapy and drug screening. In this presentation, I focus on the cell therapy research. We have developed a differentiation induction method that mimics the developmental stages and have succeeded in inducing skeletal muscle stem cells that are applicable to cell transplantation therapy. We have found that cell transplantation into Duchenne muscular dystrophy (DMD) model mice is effective in regenerating more than 10% of dystrophin-positive fibers. In addition, some of the cells have been engrafted as satellite cells in vivo, and it is expected that the therapeutic effect will continue for a long period of time. As for the efficacy to the motor function, we have recently revealed that the regeneration of dystrophin positive myofibers in DMD model mice mainly ameliorates muscle fatigue tolerance rather than maximal contraction force in vivo. We have also developed a differentiation method to induce mesenchymal stromal cells (MSCs) from iPSCs. Transplantation of iPSC-derived MSCs (iMSCs) into Ullrich congenital muscular dystrophy (UCMD) model mice enabled the restoratio
What this study does not prove
- • This study does not prove SVF is an approved treatment or a replacement for standard care.
- • This is preclinical work; animal or laboratory results cannot be applied to humans.
Evidence level
Evidence from laboratory and animal studies provides groundwork for understanding mechanisms and potential before human studies continue.
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