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Title: Induced Fetal Human Muscle Stem Cells with High Therapeutic Potential in a Mouse Muscular Dystrophy Model
Authors: Zhao, Mingming
Tazumi, Atsutoshi
Takayama, Satoru
Takenaka-Ninagawa, Nana
Nalbandian, Minas
Nagai, Miki
Nakamura, Yumi
Nakasa, Masanori
Watanabe, Akira
Ikeya, Makoto  kyouindb  KAKEN_id
Hotta, Akitsu  kyouindb  KAKEN_id  orcid (unconfirmed)
Ito, Yuta
Sato, Takahiko
Sakurai, Hidetoshi  kyouindb  KAKEN_id  orcid (unconfirmed)
Author's alias: 趙, 明明
田積, 充年
高山, 了
竹中, 菜々
中村, 友美
中佐, 昌紀
渡辺, 亮
池谷, 真
堀田, 秋津
伊東, 佑太
佐藤, 貴彦
櫻井, 英俊
Keywords: muscular dystrophy
pluripotent stem cells
muslce stem cells
myogenic differentiation
WNT agonist
Issue Date: 14-Jul-2020
Publisher: Elsevier BV
Journal title: Stem cell report
Volume: 15
Issue: 1
Start page: 80
End page: 94
Abstract: Duchenne muscular dystrophy (DMD) is a progressive and fatal muscle-wasting disease caused by DYSTROPHIN deficiency. Cell therapy using muscle stem cells (MuSCs) is a potential cure. Here, we report a differentiation method to generate fetal MuSCs from human induced pluripotent stem cells (iPSCs) by monitoring MYF5 expression. Gene expression profiling indicated that MYF5-positive cells in the late stage of differentiation have fetal MuSC characteristics, while MYF5-positive cells in the early stage of differentiation have early myogenic progenitor characteristics. Moreover, late-stage MYF5-positive cells demonstrated good muscle regeneration potential and produced DYSTROPHIN in vivo after transplantation into DMD model mice, resulting in muscle function recovery. The engrafted cells also generated PAX7-positive MuSC-like cells under the basal lamina of DYSTROPHIN-positive fibers. These findings suggest that MYF5-positive fetal MuSCs induced in the late stage of iPSC differentiation have cell therapy potential for DMD.
Description: 筋ジストロフィーモデルマウスにおけるヒトiPS細胞由来骨格筋幹細胞の移植効果を確認. 京都大学プレスリリース. 2020-07-20.
Rights: © 2020 The Authors. This is an open access article under the CC BY-NC-ND license (
DOI(Published Version): 10.1016/j.stemcr.2020.06.004
PubMed ID: 32619494
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