Inherited SLP76 deficiency in humans causes severe combined immunodeficiency, neutrophil and platelet defects.


Journal

The Journal of experimental medicine
ISSN: 1540-9538
Titre abrégé: J Exp Med
Pays: United States
ID NLM: 2985109R

Informations de publication

Date de publication:
01 03 2021
Historique:
received: 24 05 2020
revised: 06 09 2020
accepted: 08 10 2020
entrez: 24 11 2020
pubmed: 25 11 2020
medline: 15 9 2021
Statut: ppublish

Résumé

The T cell receptor (TCR) signaling pathway is an ensemble of numerous proteins that are crucial for an adequate immune response. Disruption of any protein involved in this pathway leads to severe immunodeficiency and unfavorable clinical outcomes. Here, we describe an infant with severe immunodeficiency who was found to have novel biallelic mutations in SLP76. SLP76 is a key protein involved in TCR signaling and in other hematopoietic pathways. Previous studies of this protein were performed using Jurkat-derived human leukemic T cell lines and SLP76-deficient mice. Our current study links this gene, for the first time, to a human immunodeficiency characterized by early-onset life-threatening infections, combined T and B cell immunodeficiency, severe neutrophil defects, and impaired platelet aggregation. Hereby, we characterized aspects of the patient's immune phenotype, modeled them with an SLP76-deficient Jurkat-derived T cell line, and rescued some consequences using ectopic expression of wild-type SLP76. Understanding human diseases due to SLP76 deficiency is helpful in explaining the mixed T cell and neutrophil defects, providing a guide for exploring human SLP76 biology.

Identifiants

pubmed: 33231617
pii: 211562
doi: 10.1084/jem.20201062
pmc: PMC7690938
pii:
doi:

Substances chimiques

Adaptor Proteins, Signal Transducing 0
Phosphoproteins 0
Receptors, Antigen, B-Cell 0
Receptors, Antigen, T-Cell 0
SLP-76 signal Transducing adaptor proteins 0

Types de publication

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

Langues

eng

Sous-ensembles de citation

IM

Informations de copyright

© 2020 Lev et al.

Déclaration de conflit d'intérêts

Disclosures: The authors declare no competing interests exist.

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Auteurs

Atar Lev (A)

Pediatric Department A and Immunology Service, Jeffrey Modell Foundation Center, Edmond and Lily Safra Children's Hospital, Sheba Medical Center, Tel Hashomer, Israel.
The Mina and Everard Goodman Faculty of Life Sciences, Advanced Materials and Nanotechnology Institute, Bar-Ilan University, Ramat Gan, Israel.

Yu Nee Lee (YN)

Pediatric Department A and Immunology Service, Jeffrey Modell Foundation Center, Edmond and Lily Safra Children's Hospital, Sheba Medical Center, Tel Hashomer, Israel.

Guangping Sun (G)

Laboratory of Allergic Diseases, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD.

Enas Hallumi (E)

Department of Immunology, Ruth and Bruce Rappaport Faculty of Medicine, Technion-Israel Institute of Technology, Haifa, Israel.

Amos J Simon (AJ)

Pediatric Department A and Immunology Service, Jeffrey Modell Foundation Center, Edmond and Lily Safra Children's Hospital, Sheba Medical Center, Tel Hashomer, Israel.
Division of Haematology and Bone Marrow Transplantation, Sheba Medical Center, Tel Hashomer, Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.

Keren S Zrihen (KS)

Pediatric Department A and Immunology Service, Jeffrey Modell Foundation Center, Edmond and Lily Safra Children's Hospital, Sheba Medical Center, Tel Hashomer, Israel.

Shiran Levy (S)

Pediatric Department A and Immunology Service, Jeffrey Modell Foundation Center, Edmond and Lily Safra Children's Hospital, Sheba Medical Center, Tel Hashomer, Israel.

Tal Beit Halevi (T)

Pediatric Department A and Immunology Service, Jeffrey Modell Foundation Center, Edmond and Lily Safra Children's Hospital, Sheba Medical Center, Tel Hashomer, Israel.

Maria Papazian (M)

Pediatric Department A and Immunology Service, Jeffrey Modell Foundation Center, Edmond and Lily Safra Children's Hospital, Sheba Medical Center, Tel Hashomer, Israel.

Neta Shwartz (N)

Pediatric Department A and Immunology Service, Jeffrey Modell Foundation Center, Edmond and Lily Safra Children's Hospital, Sheba Medical Center, Tel Hashomer, Israel.

Ido Somekh (I)

Department of Pediatric Hematology Oncology, Schneider Children's Medical Center of Israel, Petah Tikva, Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.

Sarina Levy-Mendelovich (S)

The Israeli National Hemophilia Center and Thrombosis Unit, The Amalia Biron Research Institute of Thrombosis and Hemostasis, Sheba Medical Center, Tel Hashomer, Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.

Baruch Wolach (B)

Department of Pediatrics and Laboratory for Leukocyte Function, Meir Medical Center, Kfar Saba, Israel.

Ronit Gavrieli (R)

Department of Pediatrics and Laboratory for Leukocyte Function, Meir Medical Center, Kfar Saba, Israel.

Helly Vernitsky (H)

Division of Haematology and Bone Marrow Transplantation, Sheba Medical Center, Tel Hashomer, Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.

Ortal Barel (O)

The Genomic Unit, Sheba Cancer Research Center, Sheba Medical Center, Tel Hashomer, Israel.
Cancer Research Center, Wohl Institute for Translational Medicine, Sheba Medical Center, Tel Hashomer, Israel.

Elisheva Javasky (E)

The Genomic Unit, Sheba Cancer Research Center, Sheba Medical Center, Tel Hashomer, Israel.
Cancer Research Center, Wohl Institute for Translational Medicine, Sheba Medical Center, Tel Hashomer, Israel.

Tali Stauber (T)

Pediatric Department A and Immunology Service, Jeffrey Modell Foundation Center, Edmond and Lily Safra Children's Hospital, Sheba Medical Center, Tel Hashomer, Israel.

Chi A Ma (CA)

Laboratory of Allergic Diseases, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD.

Yuan Zhang (Y)

Laboratory of Allergic Diseases, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD.
Department of Pediatrics, Columbia University Irving Medical Center, New York, NY.

Ninette Amariglio (N)

The Mina and Everard Goodman Faculty of Life Sciences, Advanced Materials and Nanotechnology Institute, Bar-Ilan University, Ramat Gan, Israel.
Cancer Research Center, Sheba Medical Center, Tel Hashomer, Ramat Gan, Israel.

Gideon Rechavi (G)

Cancer Research Center, Wohl Institute for Translational Medicine, Sheba Medical Center, Tel Hashomer, Israel.
Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.

Ayal Hendel (A)

The Mina and Everard Goodman Faculty of Life Sciences, Advanced Materials and Nanotechnology Institute, Bar-Ilan University, Ramat Gan, Israel.

Deborah Yablonski (D)

Department of Immunology, Ruth and Bruce Rappaport Faculty of Medicine, Technion-Israel Institute of Technology, Haifa, Israel.

Joshua D Milner (JD)

Laboratory of Allergic Diseases, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD.
Department of Pediatrics, Columbia University Irving Medical Center, New York, NY.

Raz Somech (R)

Pediatric Department A and Immunology Service, Jeffrey Modell Foundation Center, Edmond and Lily Safra Children's Hospital, Sheba Medical Center, Tel Hashomer, Israel.
Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.

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