Application of differentiated human tonsil–derived stem cells to trembler-J mice

Saeyoung Park, Yoonyoung Choi, Geon Kwak, Young Bin Hong, Namhee Jung, Jieun Kim, Byung Ok Choi, Sung Chul Jung

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7 Scopus citations


Introduction: Mesenchymal stem cells (MSCs) can differentiate into various cell types. Methods: In this study we investigated the potential of human tonsil–derived MSCs (T-MSCs) for neuromuscular regeneration in trembler-J (Tr-J) mice, a model for Charcot–Marie–Tooth disease type 1A (CMT1A). Results: T-MSCs differentiated toward skeletal myocytes with increased expression of skeletal muscle–related markers (including troponin I type 1, and myogenin), and the formation of myotubes in vitro. In-situ transplantation of T-MSC–derived myocytes (T-MSC myocytes) into the gastrocnemius muscle in Tr-J mice enhanced motor function, with recovery of compound muscle action potential amplitudes. Morphology of the sciatic nerve and skeletal muscle recovered without the formation of teratomas, and the expression levels of nerve growth factor and glial-cell-line–derived neurotrophic factor were increased significantly in T-MSC myocytes compared with T-MSCs in vitro. Discussion: Transplantation of T-MSC myocytes could enable neuromuscular regeneration in patients with CMT1A. Muscle Nerve 57: 478–486, 2018.

Original languageEnglish
Pages (from-to)478-486
Number of pages9
JournalMuscle and Nerve
Issue number3
StatePublished - Mar 2018

Bibliographical note

Funding Information:
Additional supporting information may be found in the online version of this article Abbreviations: ANOVA, analysis of variance; ASC, adipose tissue– derived mesenchymal stem cell; BM-MSC, bone marrow–derived mesenchymal stem cell; CMAP, compound muscle action potential; CMT, Charcot–Marie–Tooth; CMT1A, Charcot–Marie–Tooth disease type 1A; DAPI, 4′,6-diamidino-2-phenylindole; DMEM, Dulbecco’s modified Eagle medium; FBS, fetal bovine serum; GDNF, glial-cell line–derived neurotrophic factor; IGF-1, insulin-like growth factor 1; IgG, immunoglobulin G; iPSCs, induced pluripotent stem cells; MBP, myelin basic protein; MNCV, motor nerve conduction velocity; MSC, mesenchymal stem cell; NCS, nerve conduction study; NF-H, neurofilament heavy polypeptide; NGF, nerve growth factor; PFA, paraformaldehyde; PMP-22, peripheral myelin protein 22; TGF-β, transforming growth factor-beta; T-MSC, tonsil-derived MSC; T-MSC myocyte, T-MSC–derived myocyte; TNNI-1, troponin I type 1; Tr-J, trembler-J; TST, tail suspension test; VEGF, vascular endothelial growth factor Key words: Charcot–Marie–Tooth disease; neuromuscular; regeneration; stem cell therapy; tonsil-derived MSCs; trembler-J mice This work was supported by a grant from the Ministry of Health and Welfare, Republic of Korea (Korean Health Technology R&D Project HI12C0135).

Publisher Copyright:
© 2017 Wiley Periodicals, Inc.


  • Charcot–Marie–Tooth disease
  • neuromuscular
  • regeneration
  • stem cell therapy
  • tonsil-derived MSCs
  • trembler-J mice


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