ORIGINAL RESEARCH article
Front. Neurol.
Sec. Neuromuscular Disorders and Peripheral Neuropathies
Myostatin Inhibition with Orally Administered Lactobacillus casei Expressing a Modified Human Myostatin Protein: Functional Benefits and Translational Potential in Advanced Duchenne Muscular Dystrophy
Provisionally accepted
- 1School of Medicine, Sungkyunkwan University, Suwon, Republic of Korea
- 2Samsung Medical Center, Gangnam-gu, Republic of Korea
- 3MOA Life Plus Co. Ltd, Yongin, Republic of Korea
- 4Future & Tech Corp, Daejeon, Republic of Korea
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Background: Duchenne muscular dystrophy (DMD) is a progressive neuromuscular disorder that requires novel therapeutic approaches beyond dystrophin restoration. Myostatin, a negative regulator of muscle growth, has emerged as a promising target to enhance muscle mass and function. Methods: We evaluated the efficacy of an orally administered Lactobacillus casei strain expressing a modified human myostatin protein (BLS-M22), in 32-week-old mdx mice. Animals received BLS-M22 or control (L. casei-pgsA) for 8 weeks (control group = 8, treated group = 7) and 12 weeks (control group = 8, treated group = 9). Results: BLS-M22 elicited a robust systemic anti-myostatin antibody response and significantly reduced serum creatine kinase levels, indicating attenuated muscle damage. Treated mice showed improved endurance in rotarod performance. However, no significant differences were observed in body weight, muscle fiber cross-sectional area, or fibrosis, reflecting the limited regenerative capacity at an advanced disease stage. Conclusion: This study demonstrates that myostatin inhibition with orally administered Lactobacillus casei expressing a modified human myostatin protein confers functional benefits even in advanced DMD, while highlighting its therapeutic limitations without concomitant dystrophin restoration. As a cost-effective, non-invasive, and immunologically distinct platform, this system holds translational potential not only for DMD but also for broader applications in sarcopenia and metabolic disorders.
Keywords: Duchenne muscular dystrophy, Myostatin inhibition, oral immunotherapy, Lactobacillus casei, aged mdx mouse model, PgsA-based antigen display
Received: 29 Aug 2025; Accepted: 21 Nov 2025.
Copyright: © 2025 Lee, Kim, Yena, Kim and LEE. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence: JEEHUN LEE
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