Long-term robustness of a T-cell system emerging from somatic rescue of a genetic block in T-cell development.


Journal

EBioMedicine
ISSN: 2352-3964
Titre abrégé: EBioMedicine
Pays: Netherlands
ID NLM: 101647039

Informations de publication

Date de publication:
Sep 2020
Historique:
received: 14 05 2020
revised: 03 08 2020
accepted: 04 08 2020
pubmed: 26 8 2020
medline: 29 6 2021
entrez: 26 8 2020
Statut: ppublish

Résumé

The potential of a single progenitor cell to establish and maintain long-term protective T-cell immunity in humans is unknown. For genetic disorders disabling T-cell immunity, somatic reversion was shown to support limited T-cell development attenuating the clinical phenotype. However, the cases reported so far deteriorated over time leaving unanswered the important question of long-term activity of revertant precursors and the robustness of the resulting T-cell system. We applied TCRβ-CDR3 sequencing and mass cytometry on serial samples of a now 18 year-old SCIDX1 patient with somatic reversion to analyse the longitudinal diversification and stability of a T-cell system emerging from somatic gene rescue. We detected close to 10 Genetic correction in the appropriate cell type, in our patient most likely in a T-cell biased self-renewing hematopoietic progenitor, can yield a diverse T-cell system that provides long-term repertoire stability, does not show evidence for progressive exhaustion and is capable of providing protective and regulated T-cell immunity for at least two decades. DFG EH 145/9-1, DFG SCHW 432/4-1 and the German Research Foundation under Germany's Excellence Strategy-EXC-2189-Project ID: 390939984.

Identifiants

pubmed: 32841837
pii: S2352-3964(20)30337-6
doi: 10.1016/j.ebiom.2020.102961
pmc: PMC7452388
pii:
doi:

Substances chimiques

Biomarkers 0

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

102961

Informations de copyright

Copyright © 2020 The Author(s). Published by Elsevier B.V. All rights reserved.

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Auteurs

Patrick Kury (P)

Institute for Immunodeficiency, Center for Chronic Immunodeficiency (CCI), Medical Center - University of Freiburg, Faculty of Medicine, University of Freiburg, Breisacher Straße 115, 79106 Freiburg, Germany; Faculty of Biology, University of Freiburg, Schaenzlestrasse 1, D-79104 Freiburg, Germany.

Marita Führer (M)

Institute for Clinical Transfusion Medicine and Immunogenetics Ulm, German Red Cross Blood Service, Baden-Wuerttemberg - Hessen, Ulm, Germany.

Sebastian Fuchs (S)

Roche Pharma Research and Early Development, Immunology, Infectious Diseases and Ophthalmology (I2O) Discovery and Translational Area, Roche Innovation Center Basel, Basel, Switzerland.

Myriam R Lorenz (MR)

Institute for Transfusion Medicine, University of Ulm, Ulm, Germany.

Orlando Bruno Giorgetti (OB)

Department of Developmental Immunology, Max Planck Institute of Immunobiology and Epigenetics Freiburg, Freiburg, Germany.

Shahrzad Bakhtiar (S)

Division for Pediatric Stem-Cell Transplantation, Immunology and Intensive Medicine, University Hospital Frankfurt, Frankfurt/Main, Germany.

Andreas P Frei (AP)

Roche Pharma Research and Early Development, Immunology, Infectious Diseases and Ophthalmology (I2O) Discovery and Translational Area, Roche Innovation Center Basel, Basel, Switzerland.

Paul Fisch (P)

Department of Pathology, Faculty of Medicine, University of Freiburg, Freiburg, Germany.

Thomas Boehm (T)

Department of Developmental Immunology, Max Planck Institute of Immunobiology and Epigenetics Freiburg, Freiburg, Germany.

Klaus Schwarz (K)

Institute for Clinical Transfusion Medicine and Immunogenetics Ulm, German Red Cross Blood Service, Baden-Wuerttemberg - Hessen, Ulm, Germany; Institute for Transfusion Medicine, University of Ulm, Ulm, Germany.

Carsten Speckmann (C)

Institute for Immunodeficiency, Center for Chronic Immunodeficiency (CCI), Medical Center - University of Freiburg, Faculty of Medicine, University of Freiburg, Breisacher Straße 115, 79106 Freiburg, Germany; Center for Pediatrics, Department of Pediatric Hematology and Oncology, University Medical Center, Faculty of Medicine, University of Freiburg, Freiburg, Germany.

Stephan Ehl (S)

Institute for Immunodeficiency, Center for Chronic Immunodeficiency (CCI), Medical Center - University of Freiburg, Faculty of Medicine, University of Freiburg, Breisacher Straße 115, 79106 Freiburg, Germany; CIBBS -Centre for Integrative Biological Signaling Studies, University of Freiburg, Freiburg, Germany. Electronic address: stephan.ehl@uniklinik-freiburg.de.

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Classifications MeSH