Preclinical evaluation for engraftment of CD34
Anemia, Sickle Cell
/ genetics
Animals
Antigens, CD34
/ metabolism
Gene Editing
/ methods
Gene Targeting
/ methods
Hematopoietic Stem Cell Transplantation
/ methods
Hematopoietic Stem Cells
/ metabolism
Hemoglobin, Sickle
/ genetics
Heterografts
/ immunology
Humans
Macaca mulatta
/ genetics
Mice
RNA, Guide, Kinetoplastida
/ metabolism
beta-Globins
/ genetics
CRISPR/Cas9
electroporation
gene correction
genome editing
hematopoietic stem cell
large animal model
non-human primate
sickle cell disease
transplantation
β-globin gene
Journal
Cell reports. Medicine
ISSN: 2666-3791
Titre abrégé: Cell Rep Med
Pays: United States
ID NLM: 101766894
Informations de publication
Date de publication:
20 04 2021
20 04 2021
Historique:
received:
28
05
2020
revised:
27
07
2020
accepted:
19
03
2021
entrez:
5
5
2021
pubmed:
6
5
2021
medline:
6
5
2021
Statut:
epublish
Résumé
Sickle cell disease (SCD) is caused by a 20A > T mutation in the β-globin gene. Genome-editing technologies have the potential to correct the SCD mutation in hematopoietic stem cells (HSCs), producing adult hemoglobin while simultaneously eliminating sickle hemoglobin. Here, we developed high-efficiency viral vector-free non-footprint gene correction in SCD CD34
Identifiants
pubmed: 33948577
doi: 10.1016/j.xcrm.2021.100247
pii: S2666-3791(21)00063-X
pmc: PMC8080237
doi:
Substances chimiques
Antigens, CD34
0
Hemoglobin, Sickle
0
RNA, Guide
0
beta-Globins
0
Types de publication
Journal Article
Research Support, N.I.H., Intramural
Research Support, Non-U.S. Gov't
Langues
eng
Pagination
100247Déclaration de conflit d'intérêts
L.L., C.A., and M.V.P. were employees at MaxCyte during the period of this work.
Références
Bone Marrow Transplant. 2001 Jun;27(11):1201-9
pubmed: 11551032
Nat Biotechnol. 2018 Jan;36(1):95-102
pubmed: 29176614
Mol Ther Methods Clin Dev. 2018 Mar 22;9:247-256
pubmed: 29766032
Gene Ther. 2014 Feb;21(2):148-57
pubmed: 24257347
JAMA. 2014 Jul 2;312(1):48-56
pubmed: 25058217
Stem Cells. 2017 Mar;35(3):586-596
pubmed: 27739611
Sci Transl Med. 2019 Jul 31;11(503):
pubmed: 31366580
Blood. 1995 Jun 1;85(11):3048-57
pubmed: 7538814
Mol Ther. 2019 Sep 4;27(9):1586-1596
pubmed: 31253582
Mol Ther. 2012 Oct;20(10):1882-92
pubmed: 22871664
Blood. 1984 Apr;63(4):921-6
pubmed: 6200161
Nat Rev Genet. 2014 May;15(5):321-34
pubmed: 24690881
Mol Ther Methods Clin Dev. 2021 Mar 03;21:121-132
pubmed: 33816645
Mol Ther Methods Clin Dev. 2019 Jan 25;13:187-196
pubmed: 30788387
Mol Ther Nucleic Acids. 2019 Sep 6;17:277-288
pubmed: 31279229
Biochem Biophys Res Commun. 1973 Aug 21;53(4):1240-5
pubmed: 4748816
Hum Gene Ther. 1999 Aug 10;10(12):1953-64
pubmed: 10466629
Nat Rev Immunol. 2007 Feb;7(2):118-30
pubmed: 17259968
J Virol. 2009 Oct;83(19):9854-62
pubmed: 19625395
Mol Ther Nucleic Acids. 2013 Sep 17;2:e122
pubmed: 24045711
Science. 2013 Oct 11;342(6155):253-7
pubmed: 24115442
Nucleic Acids Res. 2019 Sep 5;47(15):7955-7972
pubmed: 31147717
Sci Transl Med. 2017 Jan 11;9(372):
pubmed: 28077679
N Engl J Med. 1999 Apr 1;340(13):1021-30
pubmed: 10099145
Nature. 2016 Nov 17;539(7629):384-389
pubmed: 27820943
N Engl J Med. 2011 Sep 1;365(9):807-14
pubmed: 21879898
Proc Natl Acad Sci U S A. 2020 Jun 30;117(26):15018-15027
pubmed: 32527859
Gene Ther. 2011 Nov;18(11):1078-86
pubmed: 21544097
Nat Commun. 2019 Oct 2;10(1):4479
pubmed: 31578323
Cell Rep. 2020 Sep 1;32(9):108093
pubmed: 32877675
N Engl J Med. 2017 Mar 2;376(9):848-855
pubmed: 28249145
Blood. 2011 Jan 20;117(3):798-807
pubmed: 21041718
N Engl J Med. 2018 Jul 19;379(3):226-235
pubmed: 30021096
Blood. 1996 Dec 15;88(12):4701-10
pubmed: 8977264
Sci Transl Med. 2016 Oct 12;8(360):360ra134
pubmed: 27733558
PLoS One. 2017 Jan 6;12(1):e0169774
pubmed: 28060945
Blood. 2017 Oct 26;130(17):1946-1948
pubmed: 28887325
Mol Ther Methods Clin Dev. 2016 Sep 14;3:16059
pubmed: 27652288
Blood. 2013 Apr 18;121(16):3216-27
pubmed: 23327923
Haematologica. 2020 Feb 13;105(10):e497
pubmed: 33054091
Sci Rep. 2018 Jul 19;8(1):10950
pubmed: 30026539
Cell Transplant. 2010;19(11):1465-73
pubmed: 21214970
Mol Ther. 2018 Feb 7;26(2):468-479
pubmed: 29221806
N Engl J Med. 2009 Dec 10;361(24):2309-17
pubmed: 20007560
Cell Stem Cell. 2019 May 2;24(5):821-828.e5
pubmed: 31051134
Nat Commun. 2019 Jun 28;10(1):2866
pubmed: 31253785
Hum Gene Ther Clin Dev. 2017 Sep;28(3):136-144
pubmed: 28447889
Blood. 1998 Aug 15;92(4):1131-41
pubmed: 9694700
Mol Ther. 2019 Aug 7;27(8):1389-1406
pubmed: 31178391
N Engl J Med. 2017 Feb 2;376(5):429-439
pubmed: 27959701
Nat Biotechnol. 2019 Mar;37(3):224-226
pubmed: 30809026
Nat Cell Biol. 2016 Dec 23;19(1):1-9
pubmed: 28008184
Stem Cells Transl Med. 2020 May;9(5):590-602
pubmed: 32034898
Nat Rev Mol Cell Biol. 2018 Jan;19(1):20-30
pubmed: 29018283
Mol Ther Methods Clin Dev. 2019 Nov 11;15:430-437
pubmed: 31890735
Nat Med. 2016 Sep;22(9):987-90
pubmed: 27525524
Exp Hematol. 2011 Jul;39(7):795-805
pubmed: 21549175
Blood. 2015 Apr 23;125(17):2597-604
pubmed: 25733580