βT87Q-Globin Gene Therapy Reduces Sickle Hemoglobin Production, Allowing for
gene therapy
hemoglobin
immortalized erythrocytes
sickle cell disease
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:
29
01
2020
accepted:
13
04
2020
entrez:
15
5
2020
pubmed:
15
5
2020
medline:
15
5
2020
Statut:
epublish
Résumé
Lentiviral addition of βT87Q-globin, a modified β-globin with an anti-sickling mutation, is currently being used in gene therapy trials for sickle cell disease (SCD) and β-thalassemia patients. βT87Q-globin interferes with sickle hemoglobin (HbS) polymerization. Here, we generated the SCD mutation in an immortalized human erythroid cell line (HUDEP-2) to investigate the anti-sickling activity of βT87Q-globin. Sickle HUDEP-2 (sHUDEP-2) cells produced robust HbS after differentiation and sickled under deoxygenated conditions, comparable with SCD CD34
Identifiants
pubmed: 32405513
doi: 10.1016/j.omtm.2020.04.013
pii: S2329-0501(20)30073-5
pmc: PMC7210457
doi:
Types de publication
Journal Article
Langues
eng
Pagination
912-921Références
BMC Biol. 2013 May 30;11:59
pubmed: 23721193
Curr Gene Ther. 2010 Oct;10(5):404-12
pubmed: 20712578
Mol Ther Nucleic Acids. 2013 Sep 17;2:e122
pubmed: 24045711
Cytotherapy. 2018 Oct;20(10):1278-1287
pubmed: 30249524
Genome Biol. 2010;11(3):R25
pubmed: 20196867
Nat Immunol. 2018 Sep;19(9):942-953
pubmed: 30111894
J Phys Chem B. 2018 Dec 13;122(49):11579-11590
pubmed: 30179501
PLoS Med. 2013;10(7):e1001484
pubmed: 23874164
Nucleic Acids Res. 2019 Jan 8;47(D1):D766-D773
pubmed: 30357393
Stem Cells. 2017 Mar;35(3):586-596
pubmed: 27739611
Blood. 2015 Apr 23;125(17):2597-604
pubmed: 25733580
Elife. 2014 Dec 15;3:e04766
pubmed: 25497837
Exp Hematol. 2019 Jun;74:19-24.e4
pubmed: 31004744
Adv Protein Chem. 1990;40:63-279
pubmed: 2195851
Mol Cells. 2012 Jul;34(1):1-5
pubmed: 22610406
Bioinformatics. 2014 Apr 1;30(7):923-30
pubmed: 24227677
Mol Ther Methods Clin Dev. 2019 Jan 25;13:187-196
pubmed: 30788387
Leukemia. 2012 Mar;26(3):414-21
pubmed: 22173215
Hematology Am Soc Hematol Educ Program. 2005;:31-7
pubmed: 16304356
Br J Haematol. 1976 Mar;32(3):365-72
pubmed: 1252371
Cytotherapy. 2018 Jul;20(7):899-910
pubmed: 29859773
Ann N Y Acad Sci. 2016 Mar;1368(1):16-24
pubmed: 26695885
Bioinformatics. 2014 Aug 1;30(15):2114-20
pubmed: 24695404
Proc Natl Acad Sci U S A. 1993 Jun 1;90(11):5039-43
pubmed: 7685112
Mol Cell Biol. 1989 Apr;9(4):1426-34
pubmed: 2657395
Nat Biotechnol. 2019 Aug;37(8):907-915
pubmed: 31375807
PLoS One. 2013;8(3):e59890
pubmed: 23533656
F1000Res. 2017 Jun 12;6:870
pubmed: 28663793
Proc Natl Acad Sci U S A. 1988 Aug;85(16):6062-6
pubmed: 3413076
Nucleic Acids Res. 2015 Apr 20;43(7):e47
pubmed: 25605792
Nat Commun. 2019 Oct 2;10(1):4479
pubmed: 31578323
N Engl J Med. 2017 Mar 2;376(9):848-855
pubmed: 28249145
Oncogene. 2002 May 13;21(21):3368-76
pubmed: 12032775
Blood. 2018 Apr 26;131(17):1960-1973
pubmed: 29519807
Blood. 1999 Apr 1;93(7):2208-16
pubmed: 10090929
Nat Commun. 2017 Mar 14;8:14750
pubmed: 28290447
Nature. 2015 Nov 12;527(7577):192-7
pubmed: 26375006
Exp Hematol. 2018 Jun;62:7-16.e1
pubmed: 29524566
Science. 2001 Dec 14;294(5550):2368-71
pubmed: 11743206
Protein J. 2006 Dec;25(7-8):503-16
pubmed: 17131194
Blood. 1981 May;57(5):967-71
pubmed: 7214025