Induced Fetal Human Muscle Stem Cells with High Therapeutic Potential in a Mouse Muscular Dystrophy Model.
Animals
Biomarkers
/ metabolism
Cell Differentiation
Cell Lineage
Disease Models, Animal
Dystrophin
/ metabolism
Fetal Stem Cells
/ transplantation
Genes, Reporter
Green Fluorescent Proteins
/ metabolism
Humans
Induced Pluripotent Stem Cells
/ cytology
Mice
Muscle Development
Muscular Dystrophy, Duchenne
/ pathology
Myoblasts
/ transplantation
Myogenic Regulatory Factor 5
/ metabolism
PAX3 Transcription Factor
/ metabolism
Recovery of Function
Regeneration
Myf5
Pax7
WNT agonist
iPSC
muscular dystrophy
muslce stem cells
myogenic differentiation
pluripotent stem cells
Journal
Stem cell reports
ISSN: 2213-6711
Titre abrégé: Stem Cell Reports
Pays: United States
ID NLM: 101611300
Informations de publication
Date de publication:
14 07 2020
14 07 2020
Historique:
received:
24
06
2019
revised:
03
06
2020
accepted:
03
06
2020
pubmed:
4
7
2020
medline:
3
6
2021
entrez:
4
7
2020
Statut:
ppublish
Résumé
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.
Identifiants
pubmed: 32619494
pii: S2213-6711(20)30225-3
doi: 10.1016/j.stemcr.2020.06.004
pmc: PMC7363940
pii:
doi:
Substances chimiques
Biomarkers
0
Dystrophin
0
Myf5 protein, mouse
0
Myogenic Regulatory Factor 5
0
PAX3 Transcription Factor
0
Pax3 protein, mouse
138016-91-8
Green Fluorescent Proteins
147336-22-9
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
80-94Informations de copyright
Copyright © 2020 The Authors. Published by Elsevier Inc. All rights reserved.
Références
Nat Cell Biol. 2018 Jan;20(1):46-57
pubmed: 29255171
Cell Stem Cell. 2009 Mar 6;4(3):217-25
pubmed: 19265661
Cell Stem Cell. 2016 Dec 1;19(6):800-807
pubmed: 27641304
Stem Cell Reports. 2017 Jul 11;9(1):12-22
pubmed: 28528701
J Biophys Biochem Cytol. 1961 Feb;9:493-5
pubmed: 13768451
Clin Invest Med. 2006 Dec;29(6):378-82
pubmed: 17330454
Cell Stem Cell. 2012 May 4;10(5):610-9
pubmed: 22560081
Nature. 2008 Nov 27;456(7221):502-6
pubmed: 18806774
Nat Rev Drug Discov. 2011 Aug 01;10(8):621-37
pubmed: 21804598
Lancet Neurol. 2009 Oct;8(10):918-28
pubmed: 19713152
Nat Med. 2007 May;13(5):642-8
pubmed: 17417652
Science. 2007 Dec 21;318(5858):1917-20
pubmed: 18029452
Stem Cell Reports. 2015 Sep 8;5(3):419-34
pubmed: 26352798
PLoS Genet. 2010 Apr 01;6(4):e1000897
pubmed: 20368965
Nat Rev Genet. 2013 Jun;14(6):373-8
pubmed: 23609411
Cell. 2010 Feb 19;140(4):554-66
pubmed: 20178747
Cell Rep. 2013 Mar 28;3(3):661-70
pubmed: 23478022
EMBO Rep. 2004 Sep;5(9):872-6
pubmed: 15470384
Science. 2005 Sep 23;309(5743):2064-7
pubmed: 16141372
J Cell Sci. 2013 Apr 15;126(Pt 8):1868-80
pubmed: 23444384
Nat Med. 2008 Feb;14(2):134-43
pubmed: 18204461
Nat Protoc. 2016 Oct;11(10):1833-50
pubmed: 27583644
Methods Cell Biol. 1997;52:85-116
pubmed: 9379967
Cell. 2000 Sep 15;102(6):777-86
pubmed: 11030621
Stem Cell Reports. 2014 Sep 9;3(3):516-29
pubmed: 25241748
J Bone Miner Res. 2010 Jun;25(6):1216-33
pubmed: 20200949
Science. 2010 Aug 27;329(5995):1078-81
pubmed: 20647425
EMBO J. 1991 May;10(5):1135-47
pubmed: 2022185
Cell. 2008 Jul 11;134(1):37-47
pubmed: 18614009
Cell Rep. 2016 Jun 7;15(10):2301-2312
pubmed: 27239027
Stem Cell Res. 2009 Sep-Nov;3(2-3):157-69
pubmed: 19726261
Stem Cells Transl Med. 2016 Sep;5(9):1145-61
pubmed: 27217344
Mol Ther. 2009 Oct;17(10):1771-8
pubmed: 19623164
Nature. 1987 Dec 24-31;330(6150):754-8
pubmed: 2447503
Sci Rep. 2015 Aug 20;5:12831
pubmed: 26290039
Dev Biol. 1996 Dec 15;180(2):534-42
pubmed: 8954725
J Cell Biol. 1996 Feb;132(4):657-66
pubmed: 8647896
Nature. 2005 Jun 16;435(7044):948-53
pubmed: 15843801
Stem Cell Rev Rep. 2012 Dec;8(4):1109-19
pubmed: 23104134
J Cell Biol. 2006 Jan 2;172(1):103-13
pubmed: 16391000
Cell Rep. 2016 Mar 1;14(8):1940-52
pubmed: 26904948
Cell. 2007 Nov 30;131(5):861-72
pubmed: 18035408
J Clin Invest. 2014 Oct;124(10):4257-65
pubmed: 25157816
Stem Cell Reports. 2013 Nov 27;1(6):620-31
pubmed: 24371814
Development. 1987 Aug;100(4):587-98
pubmed: 3327671
Development. 2012 Dec;139(24):4536-48
pubmed: 23136394
Development. 2018 Aug 23;145(16):
pubmed: 30139810
Nat Commun. 2018 Jan 9;9(1):126
pubmed: 29317646
EMBO J. 2004 Aug 18;23(16):3430-9
pubmed: 15282552
Nat Biotechnol. 2015 Sep;33(9):962-9
pubmed: 26237517
Am J Hum Genet. 2002 Aug;71(2):365-74
pubmed: 12111668
Biochem Biophys Res Commun. 2005 Mar 11;328(2):507-16
pubmed: 15694376