Human DEF6 deficiency underlies an immunodeficiency syndrome with systemic autoimmunity and aberrant CTLA-4 homeostasis.


Journal

Nature communications
ISSN: 2041-1723
Titre abrégé: Nat Commun
Pays: England
ID NLM: 101528555

Informations de publication

Date de publication:
15 07 2019
Historique:
received: 31 08 2018
accepted: 29 05 2019
entrez: 17 7 2019
pubmed: 17 7 2019
medline: 19 12 2019
Statut: epublish

Résumé

Immune responses need to be controlled tightly to prevent autoimmune diseases, yet underlying molecular mechanisms remain partially understood. Here, we identify biallelic mutations in three patients from two unrelated families in differentially expressed in FDCP6 homolog (DEF6) as the molecular cause of an inborn error of immunity with systemic autoimmunity. Patient T cells exhibit impaired regulation of CTLA-4 surface trafficking associated with reduced functional CTLA-4 availability, which is replicated in DEF6-knockout Jurkat cells. Mechanistically, we identify the small GTPase RAB11 as an interactor of the guanine nucleotide exchange factor DEF6, and find disrupted binding of mutant DEF6 to RAB11 as well as reduced RAB11

Identifiants

pubmed: 31308374
doi: 10.1038/s41467-019-10812-x
pii: 10.1038/s41467-019-10812-x
pmc: PMC6629652
doi:

Substances chimiques

B7-1 Antigen 0
CTLA-4 Antigen 0
DEF6 protein, human 0
DNA-Binding Proteins 0
Guanine Nucleotide Exchange Factors 0
rab11 protein EC 3.6.1.-
rab GTP-Binding Proteins EC 3.6.5.2

Types de publication

Journal Article Research Support, N.I.H., Extramural Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

3106

Subventions

Organisme : Austrian Science Fund FWF
ID : P 29951
Pays : Austria
Organisme : NIAID NIH HHS
ID : P01 AI061093
Pays : United States
Organisme : NIAID NIH HHS
ID : T32 AI125179
Pays : United States

Commentaires et corrections

Type : ErratumIn

Références

J Clin Immunol. 2017 Nov;37(8):790-800
pubmed: 28956255
Inflamm Bowel Dis. 2015 Jan;21(1):40-7
pubmed: 25479458
J Clin Invest. 2015 Jun;125(6):2228-33
pubmed: 25893595
Proc Natl Acad Sci U S A. 1997 Aug 19;94(17):9273-8
pubmed: 9256472
Int Arch Allergy Immunol. 2016;171(1):45-53
pubmed: 27820937
Am J Hum Genet. 2012 Jun 8;90(6):986-1001
pubmed: 22608502
J Biol Chem. 2012 Sep 7;287(37):31073-84
pubmed: 22829599
Clin Immunol. 2018 Mar;188:52-57
pubmed: 29330115
Immunol Cell Biol. 2017 Oct;95(9):775-788
pubmed: 28611475
Immunol Rev. 2005 Apr;204:102-15
pubmed: 15790353
Bioinformatics. 2009 Jul 15;25(14):1754-60
pubmed: 19451168
Nature. 2014 Nov 27;515(7528):577-81
pubmed: 25428507
Immunity. 2003 Mar;18(3):403-14
pubmed: 12648457
Immunity. 1995 Nov;3(5):541-7
pubmed: 7584144
Nature. 2007 Feb 22;445(7130):936-40
pubmed: 17237761
J Immunol. 2009 Dec 1;183(11):7259-67
pubmed: 19915062
Nucleic Acids Res. 2017 Jul 3;45(W1):W567-W572
pubmed: 28520890
Prog Nucleic Acid Res Mol Biol. 2002;71:391-444
pubmed: 12102558
J Biol Chem. 2009 May 1;284(18):11882-91
pubmed: 19251698
Nat Med. 2014 Dec;20(12):1410-1416
pubmed: 25329329
Gut. 2015 Dec;64(12):1889-97
pubmed: 25367873
J Gastroenterol Hepatol. 2012 Jul;27(7):1159-66
pubmed: 22414241
Nature. 2015 Oct 1;526(7571):82-90
pubmed: 26367797
Nat Rev Microbiol. 2014 Sep;12(9):624-34
pubmed: 25118884
Biol Open. 2014 Dec 19;4(1):86-94
pubmed: 25527643
Nat Immunol. 2016 Dec;17(12):1352-1360
pubmed: 27776107
J Immunol. 1999 Aug 15;163(4):1868-79
pubmed: 10438921
J Immunol. 2007 Sep 15;179(6):3831-40
pubmed: 17785820
J Biol Chem. 2012 Mar 16;287(12):9429-40
pubmed: 22262842
Nat Rev Immunol. 2014 Jul;14(7):447-62
pubmed: 24948366
Nat Genet. 2016 Mar;48(3):323-30
pubmed: 26808113
J Exp Med. 2014 Oct 20;211(11):2137-49
pubmed: 25311508
Science. 1995 Nov 10;270(5238):985-8
pubmed: 7481803
Nature. 2016 Aug 17;536(7616):285-91
pubmed: 27535533
J Biol Chem. 2003 Oct 31;278(44):43541-9
pubmed: 12923183
Nucleic Acids Res. 2014 Jan;42(Database issue):D1253-60
pubmed: 24225319
Immunol Cell Biol. 2017 Oct;95(9):789-802
pubmed: 28652580
Intractable Rare Dis Res. 2014 Feb;3(1):8-11
pubmed: 25343120
Curr Opin Immunol. 2010 Dec;22(6):753-60
pubmed: 20869862
J Allergy Clin Immunol. 2012 Aug;130(2):481-8.e2
pubmed: 22721650
Science. 2014 Sep 26;345(6204):1623-1627
pubmed: 25213377
J Allergy Clin Immunol. 2012 Dec;130(6):1428-32
pubmed: 22981790
Annu Rev Immunol. 2016 May 20;34:121-49
pubmed: 26735698
Hum Immunol. 2003 Apr;64(4):389-401
pubmed: 12651066
Science. 2015 Jul 24;349(6246):436-40
pubmed: 26206937
Blood. 2017 Mar 16;129(11):1458-1468
pubmed: 28159733
J Clin Invest. 2007 Aug;117(8):2164-75
pubmed: 17657315
Nat Genet. 2014 Mar;46(3):310-5
pubmed: 24487276
Bioinformatics. 2010 Aug 15;26(16):2069-70
pubmed: 20562413
J Cell Sci. 2015 Dec 1;128(23):4341-52
pubmed: 26483383
Nat Methods. 2010 Apr;7(4):248-9
pubmed: 20354512
Cytometry B Clin Cytom. 2010 Nov;78(6):372-81
pubmed: 20533385
J Clin Invest. 2006 Mar;116(3):703-14
pubmed: 16470246
Immunol Rev. 2009 Nov;232(1):319-33
pubmed: 19909373
J Microsc. 2006 Dec;224(Pt 3):213-32
pubmed: 17210054
J Immunol. 2013 Jan 1;190(1):174-83
pubmed: 23197258
Mol Vis. 2012;18:1858-64
pubmed: 22815639
J Immunol. 1997 Jul 1;159(1):144-51
pubmed: 9200449
Adv Immunol. 2014;124:95-136
pubmed: 25175774
Sci Rep. 2016 Nov 08;6:36568
pubmed: 27824136
Phys Rev A. 1996 Nov;54(5):3863-3869
pubmed: 9913933
Immunity. 2008 Dec 19;29(6):899-911
pubmed: 19062315
Trends Immunol. 2015 Feb;36(2):63-70
pubmed: 25582039
Trends Cell Biol. 2006 Oct;16(10):522-9
pubmed: 16949823
Science. 2011 Apr 29;332(6029):600-3
pubmed: 21474713
PLoS Comput Biol. 2013;9(7):e1003153
pubmed: 23874191
Nat Protoc. 2009;4(7):1073-81
pubmed: 19561590
Cytometry A. 2015 Jan;87(1):81-5
pubmed: 25132325

Auteurs

Nina K Serwas (NK)

Ludwig Boltzmann Institute for Rare and Undiagnosed Diseases, Vienna, Austria.
CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences, Vienna, Austria.
Department of Pathology, University of California San Francisco, San Francisco, CA, USA.

Birgit Hoeger (B)

Ludwig Boltzmann Institute for Rare and Undiagnosed Diseases, Vienna, Austria.
CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences, Vienna, Austria.
St. Anna Children's Cancer Research Institute (CCRI), Vienna, Austria.

Rico C Ardy (RC)

Ludwig Boltzmann Institute for Rare and Undiagnosed Diseases, Vienna, Austria.
CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences, Vienna, Austria.

Sigrun V Stulz (SV)

Centre for Hematology and Regenerative Medicine, Department of Medicine Huddinge, Karolinska Institutet, Karolinska University Hospital Huddinge, Stockholm, Sweden.

Zhenhua Sui (Z)

Division of Cell Biology, La Jolla Institute for Allergy & Immunology, La Jolla, CA, 92037, USA.

Nima Memaran (N)

Department of Pediatrics and Adolescent Medicine, Medical University of Vienna, Vienna, Austria.
Centre for Paediatrics and Adoloscent Medicine, Hannover Medical School, Hannover, Germany.

Marie Meeths (M)

Childhood Cancer Research Unit, Department of Women's and Children's Health, Karolinska Institutet, Karolinska University Hospital Solna, Stockholm, Sweden.
Clinical Genetics Unit, Department of Molecular Medicine and Surgery, and Center for Molecular Medicine, Karolinska Institutet, Karolinska University Hospital Solna, Stockholm, Sweden.

Ana Krolo (A)

Ludwig Boltzmann Institute for Rare and Undiagnosed Diseases, Vienna, Austria.
CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences, Vienna, Austria.

Özlem Yüce Petronczki (Ö)

Ludwig Boltzmann Institute for Rare and Undiagnosed Diseases, Vienna, Austria.
CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences, Vienna, Austria.

Laurène Pfajfer (L)

Ludwig Boltzmann Institute for Rare and Undiagnosed Diseases, Vienna, Austria.
CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences, Vienna, Austria.
Center for Pathophysiology of Toulouse Purpan, INSERM UMR1043, CNRS UMR5282, Paul Sabatier University, Toulouse, France.

Tie Z Hou (TZ)

Institute of Immunity and Transplantation, Division of Infection & Immunity, School of Life and Medical Sciences, University College London, Royal Free Hospital, Rowland Hill Street, London, NW3 2PF, UK.

Neil Halliday (N)

Institute of Immunity and Transplantation, Division of Infection & Immunity, School of Life and Medical Sciences, University College London, Royal Free Hospital, Rowland Hill Street, London, NW3 2PF, UK.

Elisangela Santos-Valente (E)

CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences, Vienna, Austria.

Artem Kalinichenko (A)

Ludwig Boltzmann Institute for Rare and Undiagnosed Diseases, Vienna, Austria.
CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences, Vienna, Austria.

Alan Kennedy (A)

Institute of Immunity and Transplantation, Division of Infection & Immunity, School of Life and Medical Sciences, University College London, Royal Free Hospital, Rowland Hill Street, London, NW3 2PF, UK.

Emily M Mace (EM)

Department of Pediatrics, Baylor College of Medicine and Center for Human Immunobiology, Texas Children's Hospital, Houston, TX, 77030, USA.
Columbia University Medical Center, Columbia, NY, USA.

Malini Mukherjee (M)

Department of Pediatrics, Baylor College of Medicine and Center for Human Immunobiology, Texas Children's Hospital, Houston, TX, 77030, USA.

Bianca Tesi (B)

Clinical Genetics Unit, Department of Molecular Medicine and Surgery, and Center for Molecular Medicine, Karolinska Institutet, Karolinska University Hospital Solna, Stockholm, Sweden.

Anna Schrempf (A)

CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences, Vienna, Austria.

Winfried F Pickl (WF)

Institute of Immunology, Center for Pathophysiology, Infectiology and Immunology, Medical University of Vienna, Vienna, Austria.

Joanna I Loizou (JI)

CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences, Vienna, Austria.

Renate Kain (R)

Clinical Institute of Pathology, Medical University of Vienna, Vienna, Austria.

Bettina Bidmon-Fliegenschnee (B)

Department of Pediatrics and Adolescent Medicine, Medical University of Vienna, Vienna, Austria.

Jean-Nicolas Schickel (JN)

Department of Immunobiology, Yale University School of Medicine, New Haven, CT, 06511, USA.

Salomé Glauzy (S)

Department of Immunobiology, Yale University School of Medicine, New Haven, CT, 06511, USA.

Jakob Huemer (J)

Ludwig Boltzmann Institute for Rare and Undiagnosed Diseases, Vienna, Austria.
CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences, Vienna, Austria.

Wojciech Garncarz (W)

Ludwig Boltzmann Institute for Rare and Undiagnosed Diseases, Vienna, Austria.
CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences, Vienna, Austria.

Elisabeth Salzer (E)

Ludwig Boltzmann Institute for Rare and Undiagnosed Diseases, Vienna, Austria.
CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences, Vienna, Austria.

Iro Pierides (I)

Ludwig Boltzmann Institute for Rare and Undiagnosed Diseases, Vienna, Austria.
CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences, Vienna, Austria.

Ivan Bilic (I)

CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences, Vienna, Austria.
Takeda (Shire), Vienna, Austria.

Jens Thiel (J)

Department of Rheumatology and Clinical Immunology, University Medical Center Freiburg, Freiburg, 79106, Germany.

Peter Priftakis (P)

Astrid Lindgren Children's Hospital, Karolinska University Hospital Huddinge, Stockholm, Sweden.

Pinaki P Banerjee (PP)

Department of Pediatrics, Baylor College of Medicine and Center for Human Immunobiology, Texas Children's Hospital, Houston, TX, 77030, USA.
MD Anderson Cancer Center, Houston, TX, USA.

Elisabeth Förster-Waldl (E)

Department of Pediatrics and Adolescent Medicine, Division of Neonatology, Pediatric Intensive Care and Neuropediatrics, Medical University of Vienna, Vienna, Austria.

David Medgyesi (D)

Ludwig Boltzmann Institute for Rare and Undiagnosed Diseases, Vienna, Austria.
CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences, Vienna, Austria.

Wolf-Dietrich Huber (WD)

Department of Pediatrics and Adolescent Medicine, Medical University of Vienna, Vienna, Austria.

Jordan S Orange (JS)

Department of Pediatrics, Baylor College of Medicine and Center for Human Immunobiology, Texas Children's Hospital, Houston, TX, 77030, USA.
Columbia University Medical Center, Columbia, NY, USA.

Eric Meffre (E)

Department of Immunobiology, Yale University School of Medicine, New Haven, CT, 06511, USA.

David M Sansom (DM)

Institute of Immunity and Transplantation, Division of Infection & Immunity, School of Life and Medical Sciences, University College London, Royal Free Hospital, Rowland Hill Street, London, NW3 2PF, UK.

Yenan T Bryceson (YT)

Centre for Hematology and Regenerative Medicine, Department of Medicine Huddinge, Karolinska Institutet, Karolinska University Hospital Huddinge, Stockholm, Sweden.

Amnon Altman (A)

Division of Cell Biology, La Jolla Institute for Allergy & Immunology, La Jolla, CA, 92037, USA.

Kaan Boztug (K)

Ludwig Boltzmann Institute for Rare and Undiagnosed Diseases, Vienna, Austria. kaan.boztug@ccri.at.
CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences, Vienna, Austria. kaan.boztug@ccri.at.
St. Anna Children's Cancer Research Institute (CCRI), Vienna, Austria. kaan.boztug@ccri.at.
Department of Pediatrics and Adolescent Medicine, Medical University of Vienna, Vienna, Austria. kaan.boztug@ccri.at.
St. Anna Kinderspital, Department of Pediatrics and Adolescent Medicine, Medical University of Vienna, Vienna, Austria. kaan.boztug@ccri.at.

Articles similaires

[Redispensing of expensive oral anticancer medicines: a practical application].

Lisanne N van Merendonk, Kübra Akgöl, Bastiaan Nuijen
1.00
Humans Antineoplastic Agents Administration, Oral Drug Costs Counterfeit Drugs

Smoking Cessation and Incident Cardiovascular Disease.

Jun Hwan Cho, Seung Yong Shin, Hoseob Kim et al.
1.00
Humans Male Smoking Cessation Cardiovascular Diseases Female
Humans United States Aged Cross-Sectional Studies Medicare Part C
1.00
Humans Yoga Low Back Pain Female Male

Classifications MeSH