Complications of whole-exome sequencing for causal gene discovery in primary platelet secretion defects.


Journal

Haematologica
ISSN: 1592-8721
Titre abrégé: Haematologica
Pays: Italy
ID NLM: 0417435

Informations de publication

Date de publication:
10 2019
Historique:
received: 21 08 2018
accepted: 22 02 2019
pubmed: 2 3 2019
medline: 8 7 2020
entrez: 2 3 2019
Statut: ppublish

Résumé

Primary platelet secretion defects constitute a heterogeneous group of functional defects characterized by reduced platelet granule secretion upon stimulation by different agonists. The clinical and laboratory heterogeneity of primary platelet secretion defects warrants a tailored approach. We performed a pilot study in order to develop DNA sequence analysis pipelines for gene discovery and to create a list of candidate causal genes for platelet secretion defects. Whole-exome sequencing analysis of 14 unrelated Italian patients with primary secretion defects and 16 controls was performed on Illumina HiSeq. Variant prioritization was carried out using two filtering approaches: identification of rare, potentially damaging variants in platelet candidate genes or by selecting singletons. To corroborate the results, exome sequencing was applied in a family in which platelet secretion defects and a bleeding diathesis were present. Platelet candidate gene analysis revealed gene defects in 10/14 patients, which included

Identifiants

pubmed: 30819905
pii: haematol.2018.204990
doi: 10.3324/haematol.2018.204990
pmc: PMC6886420
doi:

Types de publication

Clinical Trial Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

2084-2090

Informations de copyright

Copyright© 2019 Ferrata Storti Foundation.

Références

Blood. 2016 Jun 9;127(23):2791-803
pubmed: 27084890
Br J Haematol. 2017 Oct;179(2):308-322
pubmed: 28748566
Blood. 2016 Jun 9;127(23):2814-23
pubmed: 27095789
PLoS One. 2013;8(4):e60396
pubmed: 23565241
Platelets. 2017 Sep;28(6):611-613
pubmed: 28267383
Nucleic Acids Res. 2007 Jul;35(Web Server issue):W169-75
pubmed: 17576678
J Thromb Haemost. 2006 Apr;4(4):766-73
pubmed: 16634745
Genet Med. 2015 May;17(5):405-24
pubmed: 25741868
Arterioscler Thromb Vasc Biol. 2000 Nov;20(11):E101-6
pubmed: 11073862
Platelets. 2018 Mar;29(2):192-195
pubmed: 28762304
Br J Haematol. 1992 Oct;82(2):388-90
pubmed: 1419822
Blood. 2005 Apr 15;105(8):3185-92
pubmed: 15632203
J Thromb Haemost. 2013 Apr 10;:
pubmed: 23574625
Br J Haematol. 2017 Mar;176(5):705-720
pubmed: 27984638
J Clin Invest. 2014 Oct;124(10):4503-16
pubmed: 25244095
J Thromb Haemost. 2019 Mar;17(3):551-554
pubmed: 30614196
J Thromb Haemost. 2011 Jul;9 Suppl 1:76-91
pubmed: 21781244
Am J Clin Pathol. 2011 Sep;136(3):350-8
pubmed: 21846909
Nat Rev Genet. 2017 Oct;18(10):599-612
pubmed: 28804138
BMC Med Genomics. 2012 Feb 21;5:7
pubmed: 22353194
Bioinformatics. 2009 Aug 1;25(15):1966-7
pubmed: 19497933
Semin Thromb Hemost. 2004 Oct;30(5):525-35
pubmed: 15497095
Haemophilia. 2016 Jul;22 Suppl 5:20-4
pubmed: 27405671
BMC Med Genomics. 2014 Dec 03;7:64
pubmed: 25466818
Semin Thromb Hemost. 2009 Mar;35(2):158-67
pubmed: 19408189
Haematologica. 2018 Jan;103(1):148-162
pubmed: 28983057
Expert Rev Hematol. 2011 Apr;4(2):137-41
pubmed: 21495923
Blood. 2016 Sep 1;128(9):1282-9
pubmed: 27235135
J Thromb Haemost. 2011 Sep;9(9):1744-51
pubmed: 21726405
Platelets. 2012;23(1):7-10
pubmed: 21770862
Nat Genet. 2014 Mar;46(3):310-5
pubmed: 24487276
Haematologica. 2007 Mar;92(3):357-65
pubmed: 17339185
Haemophilia. 2006 Jul;12 Suppl 3:128-36
pubmed: 16684008
J Clin Invest. 2014 Oct;124(10):4517-28
pubmed: 25244094
Haemophilia. 2016 Jul;22 Suppl 5:42-5
pubmed: 27405675
J Thromb Haemost. 2003 Jul;1(7):1628-36
pubmed: 12871299
Purinergic Signal. 2011 Sep;7(3):333-9
pubmed: 21484091
Nature. 2014 May 29;509(7502):575-81
pubmed: 24870542
Int J Lab Hematol. 2018 May;40 Suppl 1:89-96
pubmed: 29741246
Curr Biol. 2002 Mar 19;12(6):R212-4
pubmed: 11909549
J Thromb Haemost. 2015 Apr;13(4):643-50
pubmed: 25556537

Auteurs

Marcin M Gorski (MM)

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan.
Università degli Studi di Milano, Department of Pathophysiology and Transplantation and Fondazione Luigi Villa, Milan, Italy.

Anna Lecchi (A)

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan.

Eti A Femia (EA)

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan.

Silvia La Marca (S)

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan.

Andrea Cairo (A)

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan.

Emanuela Pappalardo (E)

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan.
Università degli Studi di Milano, Department of Pathophysiology and Transplantation and Fondazione Luigi Villa, Milan, Italy.

Luca A Lotta (LA)

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan.

Andrea Artoni (A)

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan.

Flora Peyvandi (F)

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan flora.peyvandi@unimi.it.
Università degli Studi di Milano, Department of Pathophysiology and Transplantation and Fondazione Luigi Villa, Milan, Italy.

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