Aspects of Gene Therapy Products Using Current Genome-Editing Technology in Japan.
double-strand break
gene therapy
genome editing
off-target effect
on-target mutagenesis
safety
Journal
Human gene therapy
ISSN: 1557-7422
Titre abrégé: Hum Gene Ther
Pays: United States
ID NLM: 9008950
Informations de publication
Date de publication:
10 2020
10 2020
Historique:
pubmed:
1
8
2020
medline:
24
8
2021
entrez:
1
8
2020
Statut:
ppublish
Résumé
The development of genome-editing technology could lead to breakthrough gene therapy. Genome editing has made it possible to easily knock out or modify a target gene, while current gene therapy using a virus vector or plasmid hampering modification with respect to gene replacement therapies. Clinical development using these genome-editing tools is progressing rapidly. However, it is also becoming clear that there is a possibility of unintended gene sequence modification or deletion, or the insertion of undesired genes, or the selection of cells with abnormalities in the cancer suppressor gene
Identifiants
pubmed: 32731837
doi: 10.1089/hum.2020.156
pmc: PMC7585607
doi:
Types de publication
Journal Article
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
1043-1053Références
Nat Biotechnol. 2015 Feb;33(2):187-197
pubmed: 25513782
Science. 2013 Oct 11;342(6155):253-7
pubmed: 24115442
Nat Commun. 2019 Sep 30;10(1):4439
pubmed: 31570731
PLoS One. 2015 Sep 09;10(9):e0136644
pubmed: 26351841
J Biol Chem. 2014 Aug 1;289(31):21312-24
pubmed: 24907273
Nat Commun. 2017 Apr 07;8:14958
pubmed: 28387220
Nat Biotechnol. 2015 Sep;33(9):985-989
pubmed: 26121415
Genetics. 2011 Aug;188(4):773-82
pubmed: 21828278
Cell Stem Cell. 2014 Jul 3;15(1):31-6
pubmed: 24996168
Nat Methods. 2012 Jul 01;9(8):805-7
pubmed: 22751204
Nat Med. 2018 Jul;24(7):939-946
pubmed: 29892062
Nucleic Acids Res. 2015 Jun 23;43(11):5560-71
pubmed: 25964300
Nat Rev Cancer. 2018 Nov;18(11):696-705
pubmed: 30293088
Nat Biotechnol. 2008 Jul;26(7):808-16
pubmed: 18587387
Genome Med. 2016 Nov 15;8(1):120
pubmed: 27846896
Oncogene. 2001 Sep 10;20(40):5572-9
pubmed: 11607810
Mol Cell. 2012 Aug 24;47(4):497-510
pubmed: 22920291
Blood. 2017 Jul 20;130(3):285-296
pubmed: 28539325
Front Genet. 2019 Jan 07;9:691
pubmed: 30687381
Nat Biotechnol. 2018 Sep;36(8):765-771
pubmed: 30010673
Biotechnol Adv. 2017 Jan - Feb;35(1):95-104
pubmed: 28011075
Mol Ther Methods Clin Dev. 2018 Jun 15;10:105-112
pubmed: 30073181
Nat Methods. 2016 Oct;13(10):868-74
pubmed: 27595405
Sci Rep. 2017 Nov 9;7(1):15189
pubmed: 29123154
Oncotarget. 2016 Sep 13;7(37):60535-60554
pubmed: 27528034
Nature. 2015 Nov 12;527(7577):192-7
pubmed: 26375006
Cell Stem Cell. 2014 Jul 3;15(1):27-30
pubmed: 24996167
Nat Methods. 2017 Jun;14(6):607-614
pubmed: 28459458
Genome Res. 2013 Jun;23(6):1008-17
pubmed: 23478401
Genome Res. 2010 Jan;20(1):81-9
pubmed: 19952142
Nat Biotechnol. 2015 Jan;33(1):73-80
pubmed: 25357182
Adv Exp Med Biol. 2017;1016:29-42
pubmed: 29130152
Sci Rep. 2015 Jul 28;5:12281
pubmed: 26216318
Nucleic Acids Res. 2013 Aug;41(14):e141
pubmed: 23748566
Sci Rep. 2016 Aug 17;6:31666
pubmed: 27530713
Nat Med. 2015 Feb;21(2):121-31
pubmed: 25654603
Genome Res. 2009 Jul;19(7):1279-88
pubmed: 19470664
Nat Biotechnol. 2017 Dec;35(12):1179-1187
pubmed: 29131148
Science. 2011 Sep 30;333(6051):1843-6
pubmed: 21960622
Nature. 2016 Apr 20;533(7603):420-4
pubmed: 27096365
Genome Biol. 2018 Nov 6;19(1):187
pubmed: 30400938
Science. 2016 Sep 16;353(6305):
pubmed: 27492474
Genetics. 2015 Dec;201(4):1295-306
pubmed: 26482793
Nucleic Acids Res. 2013 Oct;41(19):9049-61
pubmed: 23907390
Sci Rep. 2017 Aug 24;7(1):9363
pubmed: 28839205
Nat Med. 2018 Jul;24(7):927-930
pubmed: 29892067
Genome Res. 2016 Mar;26(3):406-15
pubmed: 26786045
Science. 2014 Jan 3;343(6166):80-4
pubmed: 24336569
PLoS One. 2018 Jan 29;13(1):e0185987
pubmed: 29377933
Nature. 2019 Feb;566(7742):105-109
pubmed: 30675057
Sci Rep. 2018 Mar 27;8(1):5272
pubmed: 29588483
Cell. 2014 Oct 23;159(3):647-61
pubmed: 25307932
Science. 2013 Feb 15;339(6121):823-6
pubmed: 23287722
Cell. 1983 May;33(1):25-35
pubmed: 6380756
PLoS One. 2017 Jan 17;12(1):e0169931
pubmed: 28095454
PLoS One. 2014 Jan 20;9(1):e85755
pubmed: 24465685
Cell Stem Cell. 2014 Jul 3;15(1):12-3
pubmed: 24996165
Science. 2003 Oct 17;302(5644):415-9
pubmed: 14564000
Nat Biotechnol. 2013 Aug;31(8):688-91
pubmed: 23929339
Mol Ther. 2016 Sep;24(9):1561-9
pubmed: 27406980
Nat Methods. 2015 Mar;12(3):237-43, 1 p following 243
pubmed: 25664545
Nat Med. 2019 Feb;25(2):249-254
pubmed: 30692695
Science. 2013 Feb 15;339(6121):819-23
pubmed: 23287718
Blood. 2015 Apr 23;125(17):2597-604
pubmed: 25733580
Int J Pharm. 2015 Oct 15;494(1):180-94
pubmed: 26278489
Nat Methods. 2017 Jun;14(6):600-606
pubmed: 28459459
Nat Commun. 2014 Jun 03;5:3964
pubmed: 24888982
Blood. 2016 May 26;127(21):2546-52
pubmed: 27053530
Nature. 2016 Dec 1;540(7631):144-149
pubmed: 27851729