Tonsil-derived mesenchymal stem cells differentiated into skeletal myocytes can lead to muscle regeneration and functional recovery in a mouse model of Duchenne muscular dystrophy (DMD), according to a new study published in the journal Tissue Engineering and Regenerative Medicine

These results suggest that tonsil-derived mesenchymal stem cells could be a promising therapeutic resource for DMD and other muscle-wasting conditions, Saeyoung Park, of the Ewha Woman’s University in Seoul, Korea, and colleagues noted.

Previous work has shown that tonsil-derived mesenchymal stem cells can differentiate into skeletal muscle cells, the authors noted.

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In the present study, the researchers investigated whether these cells could improve the symptoms of DMD using a mouse model.

Read more about the symptoms of DMD

The researchers reported that mice transplanted with the tonsil-derived mesenchymal stem cell myocytes had increased expression of dystrophin and autophagy markers compared to control animals.

The team concluded that muscle regeneration and functional recovery are achieved via the activation of autophagy in transplanted animals. More precisely, when transplanted, the myocytes had a paracrine effect inducing autophagy and promoting the recovery of damaged muscles, ultimately leading to skeletal muscle regeneration, the researchers said. 

DMD is caused by a mutation in the DMD gene, which codes for the dystrophin protein. Dystrophin acts as a shock absorber and protects muscles from damage during contraction. When dystrophin is absent due to the mutation in the DMD gene, muscle cells get damaged at each contraction, and muscle tissue is replaced by scar tissue. This result is muscle weakness, which eventually affects the diaphragm and heart. Patients eventually require a ventilator to be able to breathe. The main cause of death in DMD is heart failure due to cardiomyopathy.

There is currently no treatment for the disease, only management strategies to alleviate its symptoms.


Park S, Jeong S, Nam YH, et al. Transplantation of differentiated tonsil-derived mesenchymal stem cells ameliorates murine Duchenne muscular dystrophy via autophagy activation. Tissue Eng Regen Med. Published online November 1, 2022. doi:10.1007/s13770-022-00489-7