Alternative trafficking of Weibel-Palade body proteins in CRISPR/Cas9-engineered von Willebrand factor-deficient blood outgrowth endothelial cells.
endothelial cells
gene knockout techniques
protein transport
secretory vesicles
von Willebrand factor
Journal
Research and practice in thrombosis and haemostasis
ISSN: 2475-0379
Titre abrégé: Res Pract Thromb Haemost
Pays: United States
ID NLM: 101703775
Informations de publication
Date de publication:
Oct 2019
Oct 2019
Historique:
received:
18
01
2019
revised:
24
05
2019
accepted:
10
06
2019
entrez:
19
10
2019
pubmed:
19
10
2019
medline:
19
10
2019
Statut:
epublish
Résumé
Synthesis of the hemostatic protein von Willebrand factor (VWF) drives formation of endothelial storage organelles called Weibel-Palade bodies (WPBs). In the absence of VWF, angiogenic and inflammatory mediators that are costored in WPBs are subject to alternative trafficking routes. In patients with von Willebrand disease (VWD), partial or complete absence of VWF/WPBs may lead to additional bleeding complications, such as angiodysplasia. Studies addressing the role of VWF using VWD patient-derived blood outgrowth endothelial cells (BOECs) have reported conflicting results due to the intrinsic heterogeneity of patient-derived BOECs. To generate a VWF-deficient endothelial cell model using clustered regularly interspaced short palindromic repeats (CRISPR) genome engineering of blood outgrowth endothelial cells. We used CRISPR/CRISPR-associated protein 9 editing in single-donor cord blood-derived BOECs (cbBOECs) to generate clonal Two CRISPR editing of
Sections du résumé
BACKGROUND
BACKGROUND
Synthesis of the hemostatic protein von Willebrand factor (VWF) drives formation of endothelial storage organelles called Weibel-Palade bodies (WPBs). In the absence of VWF, angiogenic and inflammatory mediators that are costored in WPBs are subject to alternative trafficking routes. In patients with von Willebrand disease (VWD), partial or complete absence of VWF/WPBs may lead to additional bleeding complications, such as angiodysplasia. Studies addressing the role of VWF using VWD patient-derived blood outgrowth endothelial cells (BOECs) have reported conflicting results due to the intrinsic heterogeneity of patient-derived BOECs.
OBJECTIVE
OBJECTIVE
To generate a VWF-deficient endothelial cell model using clustered regularly interspaced short palindromic repeats (CRISPR) genome engineering of blood outgrowth endothelial cells.
METHODS
METHODS
We used CRISPR/CRISPR-associated protein 9 editing in single-donor cord blood-derived BOECs (cbBOECs) to generate clonal
RESULTS
RESULTS
Two
CONCLUSIONS
CONCLUSIONS
CRISPR editing of
Identifiants
pubmed: 31624792
doi: 10.1002/rth2.12242
pii: S2475-0379(22)01793-9
pmc: PMC6782018
doi:
Types de publication
Journal Article
Langues
eng
Pagination
718-732Informations de copyright
© 2019 The Authors. Research and Practice in Thrombosis and Haemostasis published by Wiley Periodicals, Inc on behalf of International Society on Thrombosis and Haemostasis.
Références
Cell. 1991 Jan 25;64(2):403-13
pubmed: 1988154
Proc Natl Acad Sci U S A. 1998 Aug 4;95(16):9524-9
pubmed: 9689113
Arterioscler Thromb Vasc Biol. 2006 May;26(5):1002-7
pubmed: 16469951
PLoS One. 2012;7(6):e38399
pubmed: 22715381
Arterioscler Thromb Vasc Biol. 2018 Mar;38(3):520-528
pubmed: 29348121
Circ Res. 2015 Jul 3;117(2):121-8
pubmed: 25940550
Blood. 2002 Dec 1;100(12):4033-9
pubmed: 12393397
Blood. 2004 Jun 1;103(11):4150-6
pubmed: 14976056
J Clin Invest. 2012 Jun;122(6):1991-2005
pubmed: 22585576
Blood. 2010 Sep 23;116(12):2183-91
pubmed: 20538801
Blood. 2011 Jan 20;117(3):1071-80
pubmed: 21048155
Blood. 1993 Aug 15;82(4):1184-91
pubmed: 8353283
Arterioscler Thromb Vasc Biol. 2017 Nov;37(11):2014-2025
pubmed: 29025705
Blood. 2014 May 15;123(20):3185-94
pubmed: 24700782
Annu Rev Biochem. 1998;67:395-424
pubmed: 9759493
Blood. 2001 Sep 1;98(5):1424-8
pubmed: 11520791
Haematologica. 2019 Oct;104(10):2091-2099
pubmed: 30630984
Proc Natl Acad Sci U S A. 2001 Mar 27;98(7):4072-7
pubmed: 11274431
J Cell Sci. 2010 Sep 1;123(Pt 17):2964-75
pubmed: 20720153
Nat Biotechnol. 2016 Feb;34(2):184-191
pubmed: 26780180
J Thromb Haemost. 2005 Aug;3(8):1702-9
pubmed: 16102036
Blood. 2000 Nov 15;96(10):3322-8
pubmed: 11071623
J Thromb Haemost. 2019 Jan;17(1):6-18
pubmed: 30375718
Oxid Med Cell Longev. 2017;2017:9759735
pubmed: 29333215
Dev Cell. 2006 Feb;10(2):223-32
pubmed: 16459301
Nat Protoc. 2012 Sep;7(9):1709-15
pubmed: 22918388
J Biol Chem. 2010 Jul 30;285(31):23842-9
pubmed: 20519501
Blood. 2010 Nov 25;116(22):4712-9
pubmed: 20716766
Nat Biotechnol. 2013 Sep;31(9):827-32
pubmed: 23873081
J Thromb Haemost. 2009 Jan;7(1):217-26
pubmed: 18983508
Cardiovasc Res. 2009 Dec 1;84(3):353-60
pubmed: 19617600
Blood Coagul Fibrinolysis. 2017 Oct;28(7):521-533
pubmed: 28362648
Blood. 2011 Oct 13;118(15):4265-73
pubmed: 21803846
Arterioscler Thromb Vasc Biol. 2018 Jul;38(7):1549-1561
pubmed: 29880488
Blood. 2004 Nov 1;104(9):2752-60
pubmed: 15226175
Br J Haematol. 2013 Apr;161(2):177-82
pubmed: 23432086
J Proteome Res. 2012 May 4;11(5):2925-36
pubmed: 22468712
J Thromb Haemost. 2014 Feb;12(2):273-81
pubmed: 24283667
Genes Cells. 2012 Jun;17(6):431-8
pubmed: 22487259
J Thromb Haemost. 2014 Feb;12(2):255-60
pubmed: 24261607
Blood. 2013 Apr 4;121(14):2773-84
pubmed: 23355534
Nat Commun. 2014 Apr 28;5:3738
pubmed: 24769558
Nat Commun. 2015 Jul 30;6:7858
pubmed: 26223854
Blood. 2005 Jan 1;105(1):145-52
pubmed: 15331450
J Thromb Haemost. 2023 Jul;21(7):1802-1812
pubmed: 37011710
J Exp Med. 1998 Nov 2;188(9):1751-6
pubmed: 9802986
PLoS One. 2017 Mar 1;12(3):e0173077
pubmed: 28249049
Thromb Haemost. 2018 Jan;118(1):152-160
pubmed: 29304535
J Cell Physiol. 2019 Jun;234(6):9184-9193
pubmed: 30317623
Nat Methods. 2014 Aug;11(8):783-784
pubmed: 25075903
Blood. 2006 Jun 15;107(12):4728-36
pubmed: 16478886
Nat Immunol. 2018 Aug;19(8):828-837
pubmed: 29988089
J Exp Med. 1998 Nov 2;188(9):1757-62
pubmed: 9802987
EMBO J. 1993 Feb;12(2):749-58
pubmed: 8440262
J Thromb Haemost. 2007 Dec;5(12):2512-9
pubmed: 17883593
Mol Biol Cell. 2000 May;11(5):1829-43
pubmed: 10793155
Blood Rev. 2012 Sep;26(5):189-96
pubmed: 22721874
Blood. 2018 Jul 12;132(2):132-140
pubmed: 29866817
Blood. 2010 Feb 18;115(7):1472-4
pubmed: 19897581
Hematology Am Soc Hematol Educ Program. 2013;2013:254-60
pubmed: 24319188
Traffic. 2006 Jun;7(6):647-62
pubmed: 16683915
N Engl J Med. 2016 Nov 24;375(21):2067-2080
pubmed: 27959741
J Cell Sci. 2008 Jan 1;121(Pt 1):19-27
pubmed: 18096688
Sci Rep. 2017 Feb 15;7:42127
pubmed: 28198371
J Thromb Haemost. 2015 Oct;13(10):1854-66
pubmed: 26270243
Res Pract Thromb Haemost. 2019 Aug 01;3(4):718-732
pubmed: 31624792