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Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
Published on: January 12, 2019
Human Skeletal Muscle Cells Derived from the Orbicularis Oculi Have Regenerative Capacity for Duchenne Muscular
Yukito Yamanaka1, Nana Takenaka2, Hidetoshi Sakurai2
1Department of Ophthalmology, Kyoto Prefectural University of Medicine, Kyoto 602-8566, Japan.
Abstract:
Skeletal muscle stem cells (MuSCs) have been proposed as suitable candidates for cell therapy in muscular disorders since they exhibit good capacity for myogenic regeneration. However, for better therapeutic outcomes, it is necessary to isolate human MuSCs from a suitable tissue source with high myogenic differentiation. In this context, we isolated CD56+CD82+ cells from the extra eyelid tissue of young and aged patients, and tested in vitro myogenic differentiation potential. In the current study, myogenic cells derived from extra eyelid tissue were characterized and compared with immortalized human myogenic cells. We found that myogenic cells derived from extra eyelid tissue proliferated and differentiated myofibers in vitro, and restored DYSTROPHIN or PAX7 expression after transplantation with these cells in mice with Duchenne muscular dystrophy. Thus, human myogenic cells derived from extra eyelid tissue including the orbicularis oculi might be good candidates for stem cell-based therapies for treating muscular diseases.
Insights
Extra eyelid tissue yields myogenic cells with potential for treating muscular disorders. These cells demonstrated effective regeneration and restoration of key protein expression in Duchenne muscular dystrophy models.
Area of Science:
- Regenerative Medicine
- Cell Biology
- Musculoskeletal Disorders
Background:
- Skeletal muscle stem cells (MuSCs) are promising for cell therapy in muscular disorders due to their regenerative capacity.
- Optimizing therapeutic outcomes requires isolating human MuSCs with high myogenic differentiation potential from accessible tissue sources.
Purpose of the Study:
- To isolate and characterize myogenic cells from extra eyelid tissue.
- To evaluate the in vitro myogenic differentiation potential of these cells.
- To compare their efficacy with immortalized human myogenic cells and assess their therapeutic potential in a Duchenne muscular dystrophy model.
Main Methods:
- Isolation of CD56+CD82+ cells from extra eyelid tissue of patients.
- In vitro assessment of myogenic differentiation and proliferation.
- In vivo transplantation studies in mice with Duchenne muscular dystrophy.
Main Results:
- Myogenic cells derived from extra eyelid tissue exhibited robust proliferation and myofiber differentiation in vitro.
- Transplantation of these cells into mice with Duchenne muscular dystrophy restored DYSTROPHIN or PAX7 expression.
- These cells showed comparable or superior characteristics to immortalized human myogenic cells.
Conclusions:
- Human myogenic cells from extra eyelid tissue, including the orbicularis oculi muscle, represent a viable source for cell-based therapies.
- These cells hold significant promise for treating muscular diseases like Duchenne muscular dystrophy.
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