mRNA-mediated delivery of gene editing tools to human primary muscle stem cells.
CRISPR/Cas9
MT: DNA editing
base editing
gene editing
human muscle stem cells
mRNA delivery
muscular dystrophy
stem cell therapy
Journal
Molecular therapy. Nucleic acids
ISSN: 2162-2531
Titre abrégé: Mol Ther Nucleic Acids
Pays: United States
ID NLM: 101581621
Informations de publication
Date de publication:
14 Jun 2022
14 Jun 2022
Historique:
received:
17
09
2021
accepted:
25
02
2022
entrez:
31
3
2022
pubmed:
1
4
2022
medline:
1
4
2022
Statut:
epublish
Résumé
Muscular dystrophies are approximately 50 devastating, untreatable monogenic diseases leading to progressive muscle degeneration and atrophy. Gene correction of transplantable cells using CRISPR/Cas9-based tools is a realistic scenario for autologous cell replacement therapies to restore organ function in many genetic disorders. However, muscle stem cells have so far lagged behind due to the absence of methods to isolate and propagate them and their susceptibility to extensive
Identifiants
pubmed: 35356683
doi: 10.1016/j.omtn.2022.02.016
pii: S2162-2531(22)00044-0
pmc: PMC8931293
doi:
Types de publication
Journal Article
Langues
eng
Pagination
47-57Informations de copyright
© 2022 The Authors.
Déclaration de conflit d'intérêts
S. Spuler and A.M. are inventors on a pending patent application (2016/030371)—technology for primary human MuSC isolation and manufacturing. S. Spuler, C.S. and H.E. are co-inventors on a pending patent application (European Patent Office 21 160 696.7) relevant to this publication.
Références
Nat Commun. 2021 May 27;12(1):3275
pubmed: 34045451
J Clin Invest. 2020 Nov 2;130(11):5652-5664
pubmed: 32946430
Mol Ther. 2014 Jan;22(1):219-25
pubmed: 23831596
Front Genet. 2021 Aug 02;12:702547
pubmed: 34408774
Brain. 2009 Oct;132(Pt 10):2831-8
pubmed: 19443631
Cell. 2005 Jul 29;122(2):289-301
pubmed: 16051152
Nature. 2019 Dec;576(7785):149-157
pubmed: 31634902
CRISPR J. 2018 Jun;1:239-250
pubmed: 31021262
Nat Biotechnol. 2015 Sep;33(9):962-9
pubmed: 26237517
Development. 2011 Sep;138(17):3647-56
pubmed: 21828093
Nature. 2016 Jan 28;529(7587):490-5
pubmed: 26735016
JCI Insight. 2021 May 24;6(10):
pubmed: 33848270
Stem Cell Reports. 2018 Mar 13;10(3):1160-1174
pubmed: 29478895
Elife. 2020 Apr 01;9:
pubmed: 32234209
Stem Cell Rev Rep. 2021 Jun;17(3):878-899
pubmed: 33349909
Nat Biotechnol. 2016 Jul;34(7):752-9
pubmed: 27240197
J Biophys Biochem Cytol. 1961 Feb;9:493-5
pubmed: 13768451
Ann Neurol. 1992 Jan;31(1):46-52
pubmed: 1371910
Science. 2010 Aug 27;329(5995):1078-81
pubmed: 20647425
Nat Commun. 2020 Aug 4;11(1):3896
pubmed: 32753727
Cell Stem Cell. 2012 May 4;10(5):610-9
pubmed: 22560081
Nat Commun. 2019 Dec 18;10(1):5776
pubmed: 31852888
Nature. 2017 Nov 23;551(7681):464-471
pubmed: 29160308
Nat Biotechnol. 2019 Mar;37(3):252-258
pubmed: 30778232
Aging Cell. 2021 Jul;20(7):e13411
pubmed: 34089289
N Engl J Med. 2021 Aug 5;385(6):493-502
pubmed: 34215024
Acta Biomater. 2021 Sep 1;131:16-40
pubmed: 34153512
Nat Biotechnol. 2020 Dec;38(12):1364
pubmed: 33273735
Nat Biotechnol. 2020 Jul;38(7):883-891
pubmed: 32433547
Nat Biotechnol. 2019 Mar;37(3):224-226
pubmed: 30809026
Nature. 2016 Apr 20;533(7603):420-4
pubmed: 27096365
Development. 2011 Sep;138(17):3639-46
pubmed: 21828092
Nat Commun. 2021 Apr 23;12(1):2437
pubmed: 33893286
Science. 2016 Jan 1;351(6268):84-8
pubmed: 26628643
Nat Rev Mol Cell Biol. 2021 Nov;22(11):713-732
pubmed: 34257452
Science. 2012 Aug 17;337(6096):816-21
pubmed: 22745249
J Clin Invest. 2014 Oct;124(10):4257-65
pubmed: 25157816
Nat Biotechnol. 2021 Aug;39(8):949-957
pubmed: 34012094
Nature. 2021 May;593(7859):429-434
pubmed: 34012082
Nat Biotechnol. 2020 Jul;38(7):892-900
pubmed: 32284586
Exp Cell Res. 2008 Apr 15;314(7):1553-65
pubmed: 18308302