Systemic delivery of a DUX4-targeting antisense oligonucleotide to treat facioscapulohumeral muscular dystrophy.
ACTA1-MCM
DUX4
FLExDUX4 mouse model
antisense oligonucleotide
facioscapulohumeral muscular dystrophy
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:
03 Dec 2021
03 Dec 2021
Historique:
received:
15
12
2020
accepted:
17
09
2021
entrez:
3
11
2021
pubmed:
4
11
2021
medline:
4
11
2021
Statut:
epublish
Résumé
Facioscapulohumeral muscular dystrophy (FSHD) is one of the most prevalent skeletal muscle dystrophies. Skeletal muscle pathology in individuals with FSHD is caused by inappropriate expression of the transcription factor DUX4, which activates different myotoxic pathways. At the moment there is no molecular therapy that can delay or prevent skeletal muscle wasting in FSHD. In this study, a systemically delivered antisense oligonucleotide (ASO) targeting the DUX4 transcript was tested
Identifiants
pubmed: 34729250
doi: 10.1016/j.omtn.2021.09.010
pii: S2162-2531(21)00239-0
pmc: PMC8526479
doi:
Types de publication
Journal Article
Langues
eng
Pagination
813-827Subventions
Organisme : NINDS NIH HHS
ID : P01 NS069539
Pays : United States
Informations de copyright
© 2021 The Author(s).
Déclaration de conflit d'intérêts
The DUX4 ASO and control ASO were supplied by Ionis Pharmaceuticals. Co-authors M.J., C.A.D., and F.R. are employees of Ionis Pharmaceuticals.
Références
J Cell Mol Med. 2013 Jan;17(1):76-89
pubmed: 23206257
N Engl J Med. 2018 Feb 15;378(7):625-635
pubmed: 29443664
J Med Genet. 2012 Mar;49(3):171-8
pubmed: 22217918
Skelet Muscle. 2020 Apr 11;10(1):8
pubmed: 32278354
Nat Genet. 2017 Jun;49(6):941-945
pubmed: 28459456
Skelet Muscle. 2014 Jun 10;4:12
pubmed: 24940479
Am J Kidney Dis. 2013 Oct;62(4):796-800
pubmed: 23561896
PLoS Genet. 2010 Oct 28;6(10):e1001181
pubmed: 21060811
PLoS One. 2009 Oct 15;4(10):e7482
pubmed: 19829708
Hum Mol Genet. 2016 Apr 15;25(8):1468-78
pubmed: 26787513
PLoS One. 2011;6(10):e26820
pubmed: 22053214
Hum Mol Genet. 2015 Oct 15;24(20):5901-14
pubmed: 26246499
Proc Natl Acad Sci U S A. 2020 Jul 14;117(28):16509-16515
pubmed: 32601200
J Med Genet. 2021 Jan 12;:
pubmed: 33436523
Mol Ther Nucleic Acids. 2017 Jun 16;7:465-474
pubmed: 28624222
Sci Rep. 2016 Jul 27;6:30377
pubmed: 27461380
FASEB J. 2019 Jul;33(7):8110-8124
pubmed: 30933664
Nucleic Acid Ther. 2013 Jun;23(3):213-27
pubmed: 23692080
Cell Rep. 2014 May 8;7(3):774-84
pubmed: 24746732
J Cell Sci. 2017 Nov 1;130(21):3685-3697
pubmed: 28935672
Science. 2010 Sep 24;329(5999):1650-3
pubmed: 20724583
J Pharmacol Exp Ther. 2015 Nov;355(2):329-40
pubmed: 26330536
Neuromuscul Disord. 2003 May;13(4):322-33
pubmed: 12868502
Dev Cell. 2012 Jan 17;22(1):38-51
pubmed: 22209328
Genes (Basel). 2017 Mar 03;8(3):
pubmed: 28273791
Mol Ther. 2021 Feb 3;29(2):848-858
pubmed: 33068777
Hum Mol Genet. 2021 Jul 9;30(15):1398-1412
pubmed: 33987655
Neurology. 2020 Jun 9;94(23):e2441-e2447
pubmed: 32467133
Mol Ther. 2016 Aug;24(8):1405-11
pubmed: 27378237
Stem Cell Res. 2012 Nov;9(3):261-9
pubmed: 23010573
Skelet Muscle. 2018 Jan 12;8(1):2
pubmed: 29329560
PLoS One. 2019 Aug 20;14(8):e0220665
pubmed: 31430305
J Cell Sci. 2016 Oct 15;129(20):3816-3831
pubmed: 27744317
Neurology. 2007 Feb 20;68(8):578-82
pubmed: 17229919
Hum Mol Genet. 2020 Apr 15;29(6):1030-1043
pubmed: 32083293
Proc Natl Acad Sci U S A. 2005 Oct 25;102(43):15545-50
pubmed: 16199517
Nucleic Acids Res. 2007;35(2):687-700
pubmed: 17182632
Ann Neurol. 2013 Nov;74(5):637-47
pubmed: 23907995
Am J Hum Genet. 2016 May 5;98(5):1020-1029
pubmed: 27153398
PLoS Genet. 2013 Apr;9(4):e1003415
pubmed: 23593020
PLoS One. 2018 Feb 7;13(2):e0192657
pubmed: 29415061
EBioMedicine. 2019 Jul;45:630-645
pubmed: 31257147
Nat Genet. 2017 Jun;49(6):935-940
pubmed: 28459454
Nat Genet. 2017 Jun;49(6):925-934
pubmed: 28459457
RNA. 2019 Sep;25(9):1211-1217
pubmed: 31209064
Elife. 2015 Jan 07;4:
pubmed: 25564732
Nucleic Acids Res. 2019 Jul 9;47(12):6029-6044
pubmed: 31127296
Nat Genet. 2012 Dec;44(12):1370-4
pubmed: 23143600
Neurology. 2020 May 26;94(21):e2270-e2282
pubmed: 32139505