CRISPR-Directed Therapeutic Correction at the
CGD
CRISPR
NCF1
chronic granulomatous disease
gene editing
gene therapy
pseudogene
Journal
Molecular therapy. Methods & clinical development
ISSN: 2329-0501
Titre abrégé: Mol Ther Methods Clin Dev
Pays: United States
ID NLM: 101624857
Informations de publication
Date de publication:
12 Jun 2020
12 Jun 2020
Historique:
received:
04
04
2020
accepted:
22
04
2020
entrez:
19
5
2020
pubmed:
19
5
2020
medline:
19
5
2020
Statut:
epublish
Résumé
Resurrection of non-processed pseudogenes may increase the efficacy of therapeutic gene editing, upon simultaneous targeting of a mutated gene and its highly homologous pseudogenes. To investigate the potency of this approach for clinical gene therapy of human diseases, we corrected a pseudogene-associated disorder, the immunodeficiency p47
Identifiants
pubmed: 32420407
doi: 10.1016/j.omtm.2020.04.015
pii: S2329-0501(20)30075-9
pmc: PMC7217921
doi:
Types de publication
Journal Article
Langues
eng
Pagination
936-943Informations de copyright
© 2020 The Author(s).
Références
Brief Funct Genomics. 2013 Nov;12(6):536-47
pubmed: 23900003
Cell Rep. 2014 Nov 20;9(4):1219-27
pubmed: 25456124
Sci Transl Med. 2017 Jan 11;9(372):
pubmed: 28077679
Hum Mutat. 2006 Jun;27(6):545-52
pubmed: 16671097
Physiol Rev. 2018 Jul 1;98(3):1739-1763
pubmed: 29897292
Nucleic Acids Res. 2018 Jun 1;46(10):e58
pubmed: 29538768
J Clin Invest. 1997 Oct 15;100(8):1907-18
pubmed: 9329953
Genomics. 1989 Jan;4(1):87-96
pubmed: 2914709
Mol Ther Methods Clin Dev. 2019 Feb 10;13:274-278
pubmed: 30859112
Adv Exp Med Biol. 2018;1044:15-25
pubmed: 29956288
PLoS One. 2009;4(4):e5234
pubmed: 19381301
Nat Methods. 2012 Jul;9(7):671-5
pubmed: 22930834
Mol Ther. 2017 Feb 1;25(2):321-330
pubmed: 28153086
Mol Ther. 2015 Jan;23(1):147-57
pubmed: 25288370
Mol Ther Nucleic Acids. 2018 Mar 2;10:1-8
pubmed: 29499925
Medicine (Baltimore). 2000 May;79(3):170-200
pubmed: 10844936
Br J Pharmacol. 1994 Dec;113(4):1455-63
pubmed: 7889302
Cancer Res. 1991 Oct 1;51(19):5329-38
pubmed: 1717147
Curr Gene Ther. 2015;15(4):416-27
pubmed: 25981636
Blood. 1996 Mar 1;87(5):1663-81
pubmed: 8634410
Biomaterials. 2015 Nov;69:191-200
pubmed: 26295532
Nat Biotechnol. 2015 Feb;33(2):187-197
pubmed: 25513782
N Engl J Med. 2010 Jan 21;362(3):239-52
pubmed: 20089974
Blood. 2009 Sep 24;114(13):2619-22
pubmed: 19541821
Nat Biotechnol. 2018 Sep;36(8):765-771
pubmed: 30010673
Science. 2003 Jan 31;299(5607):682-6
pubmed: 12560545
Blood Adv. 2016 Dec 28;1(4):270-278
pubmed: 29296942
Blood. 2001 Jan 1;97(1):305-11
pubmed: 11133775
Genomics. 1999 Jun 1;58(2):138-45
pubmed: 10366445
Stem Cell Reports. 2019 Oct 8;13(4):590-598
pubmed: 31543470
Mol Ther. 2018 Feb 7;26(2):468-479
pubmed: 29221806
Nat Biotechnol. 2016 Apr;34(4):424-9
pubmed: 26950749
Nature. 2019 Dec;576(7785):149-157
pubmed: 31634902
Nat Rev Genet. 2018 Dec;19(12):770-788
pubmed: 30323312
Eur J Med Genet. 2016 Dec;59(12):634-640
pubmed: 27771473
J Allergy Clin Immunol. 2011 May;127(5):1243-52.e7
pubmed: 21376380
Methods. 2001 Dec;25(4):402-8
pubmed: 11846609
Sci Rep. 2017 Mar 13;7:44187
pubmed: 28287132
Nature. 2014 Dec 18;516(7531):423-7
pubmed: 25337876
Blood. 2011 May 26;117(21):5561-72
pubmed: 21411759
Nat Med. 2016 Sep;22(9):987-90
pubmed: 27525524
Nat Protoc. 2013 Nov;8(11):2281-2308
pubmed: 24157548
Genomics. 2001 Jun 15;74(3):333-41
pubmed: 11414761
Front Genet. 2018 Sep 04;9:368
pubmed: 30233648