CRISPR-based gene editing enables
Journal
Science advances
ISSN: 2375-2548
Titre abrégé: Sci Adv
Pays: United States
ID NLM: 101653440
Informations de publication
Date de publication:
05 2020
05 2020
Historique:
received:
08
08
2019
accepted:
20
02
2020
entrez:
5
6
2020
pubmed:
5
6
2020
medline:
9
4
2022
Statut:
epublish
Résumé
The prototypical genetic autoimmune disease is immune dysregulation polyendocrinopathy enteropathy X-linked (IPEX) syndrome, a severe pediatric disease with limited treatment options. IPEX syndrome is caused by mutations in the forkhead box protein 3 (
Identifiants
pubmed: 32494707
doi: 10.1126/sciadv.aaz0571
pii: aaz0571
pmc: PMC7202871
doi:
Substances chimiques
FOXP3 protein, human
0
Forkhead Transcription Factors
0
Types de publication
Journal Article
Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
eaaz0571Subventions
Organisme : NCATS NIH HHS
ID : UL1 TR003142
Pays : United States
Organisme : NIAID NIH HHS
ID : R21 AI123896
Pays : United States
Organisme : NHLBI NIH HHS
ID : K23 HL140081
Pays : United States
Organisme : NCI NIH HHS
ID : R21 CA170721
Pays : United States
Organisme : NCATS NIH HHS
ID : UL1 TR001085
Pays : United States
Informations de copyright
Copyright © 2020 The Authors, some rights reserved; exclusive licensee American Association for the Advancement of Science. No claim to original U.S. Government Works. Distributed under a Creative Commons Attribution NonCommercial License 4.0 (CC BY-NC).
Références
Int Rev Immunol. 2014 Mar;33(2):129-45
pubmed: 24354325
Cell Mol Immunol. 2015 Nov;12(6):780-2
pubmed: 25544500
Blood. 2009 Nov 5;114(19):4138-41
pubmed: 19738030
J Allergy Clin Immunol. 2018 Dec;142(6):1909-1921.e9
pubmed: 29705245
Sci Transl Med. 2013 Dec 11;5(215):215ra174
pubmed: 24337481
Blood. 2016 May 26;127(21):2513-22
pubmed: 26903548
Front Immunol. 2018 Nov 01;9:2411
pubmed: 30443250
N Engl J Med. 2014 Nov 20;371(21):1994-2004
pubmed: 25409372
J Allergy Clin Immunol. 2019 Jul;144(1):317-320.e8
pubmed: 30904640
Annu Rev Immunol. 2006;24:209-26
pubmed: 16551248
Mol Ther. 2008 Jan;16(1):194-202
pubmed: 17984976
J Allergy Clin Immunol. 2020 Apr;145(4):1262-1271.e13
pubmed: 31874182
Sci Transl Med. 2016 Apr 20;8(335):335ra57
pubmed: 27099176
J Biotechnol. 2015 Oct 10;211:56-65
pubmed: 26189696
Clin Immunol. 2011 Nov;141(2):169-76
pubmed: 21865090
Nucleic Acids Res. 2014 Dec 16;42(22):e168
pubmed: 25300484
N Engl J Med. 2009 Oct 22;361(17):1710-3
pubmed: 19846862
J Allergy Clin Immunol. 2010 Feb;125(2 Suppl 2):S182-94
pubmed: 20042228
Nat Biotechnol. 2015 Feb;33(2):187-197
pubmed: 25513782
J Med Genet. 2012 May;49(5):291-302
pubmed: 22581967
Eur J Immunol. 2008 May;38(5):1381-90
pubmed: 18412171
Sci Transl Med. 2018 Aug 1;10(452):
pubmed: 30068569
Nat Biotechnol. 2015 Dec;33(12):1256-1263
pubmed: 26551060
J Allergy Clin Immunol. 2018 Mar;141(3):1036-1049.e5
pubmed: 29241729
Nat Rev Immunol. 2009 Feb;9(2):83-9
pubmed: 19114986
Cell Stem Cell. 2019 Feb 7;24(2):309-317.e7
pubmed: 30639036
N Engl J Med. 1996 Nov 21;335(21):1563-7
pubmed: 8900089
Nature. 2016 Nov 17;539(7629):384-389
pubmed: 27820943
Nat Protoc. 2018 Feb;13(2):358-376
pubmed: 29370156
J Allergy Clin Immunol. 2018 Dec;142(6):1679-1695
pubmed: 30527062
Sci Rep. 2017 Nov 17;7(1):15820
pubmed: 29150659
Front Immunol. 2018 Mar 13;9:530
pubmed: 29593749
Blood. 2011 Mar 17;117(11):3076-86
pubmed: 21252091
Mol Ther Nucleic Acids. 2014 Dec 02;3:e214
pubmed: 25462530
J Clin Invest. 2005 Nov;115(11):3276-84
pubmed: 16211090
Blood. 2013 Feb 21;121(8):1265-75
pubmed: 23169781
J Clin Invest. 2006 Jun;116(6):1713-22
pubmed: 16741580
Nat Commun. 2019 Apr 9;10(1):1634
pubmed: 30967552
N Engl J Med. 2011 Dec 22;365(25):2357-65
pubmed: 22149959
Ann N Y Acad Sci. 2018 Apr;1417(1):5-22
pubmed: 26918796
Blood. 2009 May 28;113(22):5689-91
pubmed: 19478054