Restoration of Functional Full-Length Dystrophin After Intramuscular Transplantation of Foamy Virus-Transduced Myoblasts.
AC133 Antigen
/ metabolism
Animals
Biomarkers
Cell Transplantation
Cells, Cultured
Disease Models, Animal
Dystrophin
/ genetics
Fluorescent Antibody Technique
Gene Expression
Genetic Vectors
/ administration & dosage
Male
Mice
Mice, Inbred mdx
Muscular Dystrophy, Duchenne
/ genetics
Myoblasts
/ metabolism
Nitric Oxide Synthase Type I
/ metabolism
Regeneration
Sarcoglycans
/ metabolism
Spumavirus
/ genetics
Transduction, Genetic
Duchenne muscular dystrophy
codon-optimized full-length dystrophin
foamy virus
intramuscular transplantation
mdx nude mice
Journal
Human gene therapy
ISSN: 1557-7422
Titre abrégé: Hum Gene Ther
Pays: United States
ID NLM: 9008950
Informations de publication
Date de publication:
02 2020
02 2020
Historique:
pubmed:
6
12
2019
medline:
4
6
2021
entrez:
6
12
2019
Statut:
ppublish
Résumé
Stem cell therapy is a promising strategy to treat muscle diseases such as Duchenne muscular dystrophy (DMD). To avoid immune rejection of donor cells or donor-derived muscle, autologous cells, which have been genetically modified to express dystrophin, are preferable to cells derived from healthy donors. Restoration of full-length dystrophin (FL-dys) using viral vectors is extremely challenging, due to the limited packaging capacity of the vectors, but we have recently shown that either a foamy viral or lentiviral vector is able to package FL-dys open-reading frame and transduce myoblasts derived from a DMD patient. Differentiated myotubes derived from these transduced cells produced FL-dys. Here, we transplanted the foamy viral dystrophin-corrected DMD myoblasts intramuscularly into
Identifiants
pubmed: 31801386
doi: 10.1089/hum.2019.224
pmc: PMC7047098
doi:
Substances chimiques
AC133 Antigen
0
Biomarkers
0
Dystrophin
0
Sarcoglycans
0
Nitric Oxide Synthase Type I
EC 1.14.13.39
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
241-252Subventions
Organisme : Medical Research Council
ID : G0900872
Pays : United Kingdom
Organisme : Wellcome Trust
ID : 093610/Z/10/A
Pays : United Kingdom
Organisme : Department of Health
Pays : United Kingdom
Références
PLoS Genet. 2014 Jun 12;10(6):e1004431
pubmed: 24922526
Neuromuscul Disord. 2008 Aug;18(8):597-605
pubmed: 18602263
Mol Ther. 2016 Aug;24(7):1227-36
pubmed: 27133965
Cell. 2005 Jul 29;122(2):289-301
pubmed: 16051152
Mol Ther Methods Clin Dev. 2014 Jun 04;1:14020
pubmed: 26015964
Sci Rep. 2017 Dec;7(1):79
pubmed: 28250438
Proc Natl Acad Sci U S A. 2013 Jan 8;110(2):525-30
pubmed: 23185009
Neuromuscul Disord. 2007 Dec;17(11-12):913-8
pubmed: 17826093
Pharmacotherapy. 2017 Apr;37(4):492-499
pubmed: 28152217
PLoS One. 2011 Mar 09;6(3):e17454
pubmed: 21408080
J Muscle Res Cell Motil. 2019 Aug 7;:
pubmed: 31392564
J Biol Chem. 2015 Dec 4;290(49):29531-41
pubmed: 26378238
Mol Ther. 2007 Jun;15(6):1160-6
pubmed: 17426713
Brain Pathol. 1996 Jan;6(1):37-47
pubmed: 8866746
Proc Natl Acad Sci U S A. 2006 Jan 31;103(5):1498-503
pubmed: 16428288
Mol Ther. 2013 May;21(5):964-72
pubmed: 23531552
Stem Cell Reports. 2015 Sep 8;5(3):419-34
pubmed: 26352798
Hum Gene Ther. 2014 Jan;25(1):73-81
pubmed: 24152287
Hum Mol Genet. 2008 Dec 15;17(24):3909-18
pubmed: 18784278
Mol Ther Methods Clin Dev. 2016 Mar 16;3:16004
pubmed: 27722179
EMBO Mol Med. 2015 Nov 05;7(12):1513-28
pubmed: 26543057
Skelet Muscle. 2015 Apr 28;5:11
pubmed: 25949786
Sci Rep. 2016 Jan 27;6:19750
pubmed: 26813695
Mol Ther. 2014 May;22(5):1008-17
pubmed: 24569833
Stem Cells. 2009 Oct;27(10):2478-87
pubmed: 19575422
Nat Commun. 2017 Feb 14;8:14454
pubmed: 28195574
J Neuromuscul Dis. 2019;6(1):147-159
pubmed: 30614809
Expert Opin Biol Ther. 2009 Nov;9(11):1427-36
pubmed: 19743892
Dev Dyn. 1999 Nov;216(3):244-56
pubmed: 10590476
BMC Biotechnol. 2008 Apr 02;8:35
pubmed: 18384691
PLoS One. 2012;7(9):e45173
pubmed: 23028826
Neuromuscul Disord. 2004 Jan;14(1):19-23
pubmed: 14659408
Hum Mol Genet. 1995 Aug;4(8):1251-8
pubmed: 7581361
Hum Gene Ther. 2004 Nov;15(11):1109-24
pubmed: 15610611
Cell Stem Cell. 2016 Apr 7;18(4):533-40
pubmed: 26877224
Stem Cell Rev Rep. 2016 Oct;12(5):553-559
pubmed: 27435468
J Virol. 2017 Dec 14;92(1):
pubmed: 29046446
Nat Commun. 2017 Jul 25;8:16105
pubmed: 28742067
Protein Expr Purif. 2015 May;109:47-54
pubmed: 25665506
Mol Ther. 2008 Nov;16(11):1825-32
pubmed: 18766174
Sci Transl Med. 2018 Jan 24;10(425):
pubmed: 29367344
Neurology. 2011 Jan 25;76(4):346-53
pubmed: 21263136
Curr Gene Ther. 2015;15(4):395-415
pubmed: 26159373
Neuromuscul Disord. 2007 Aug;17(8):631-8
pubmed: 17588754
Hum Gene Ther. 2018 Mar;29(3):299-311
pubmed: 28793798
J Gen Virol. 2006 May;87(Pt 5):1339-47
pubmed: 16603537
Mol Ther. 2011 Jan;19(1):165-71
pubmed: 20924363
Gene Ther. 2013 Aug;20(8):868-73
pubmed: 23388702
Ann Neurol. 2012 Mar;71(3):304-13
pubmed: 22451200
Neurol Sci. 2018 Nov;39(11):1837-1845
pubmed: 30218397
Hum Mol Genet. 2015 Aug 1;24(15):4225-37
pubmed: 25935000
Mol Ther. 2017 May 3;25(5):1125-1131
pubmed: 28416280
J Neurol Sci. 1994 Apr;122(2):162-70
pubmed: 8021701
Cell Transplant. 2005;14(7):457-67
pubmed: 16285254
Nature. 2008 Nov 27;456(7221):502-6
pubmed: 18806774
Mol Ther. 2018 Feb 7;26(2):618-633
pubmed: 29221805
J Muscle Res Cell Motil. 2019 Jun;40(2):141-150
pubmed: 31289969
Mol Ther. 2018 Oct 3;26(10):2337-2356
pubmed: 30093306
Hum Gene Ther. 2003 Aug 10;14(12):1169-79
pubmed: 12908968
Sci Rep. 2017 Aug 14;7(1):8085
pubmed: 28808269
Proc Natl Acad Sci U S A. 2011 Jan 11;108(2):762-7
pubmed: 21187385
Anat Rec (Hoboken). 2011 Feb;294(2):283-6
pubmed: 21235003
Neurology. 1993 Apr;43(4):795-800
pubmed: 8469343
Neuromuscul Disord. 1997 Mar;7(2):117-25
pubmed: 9131653
Hum Mol Genet. 2019 Jul 1;28(13):2189-2200
pubmed: 30990876
Mol Ther. 2012 Feb;20(2):462-7
pubmed: 22086232
Hum Mol Genet. 1995 Aug;4(8):1245-50
pubmed: 7581360