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Germline hypomorphic CARD11 mutations in severe atopic disease

Authors :
Michael A. Weinreich
Megan A. Cooper
Pia J. Hauk
Thomas DiMaggio
Batsukh Dorjbal
Geronimo Dubra
Eric Meffre
Brian S. Kim
Jonathan J. Lyons
Jordan K. Abbott
Erwin W. Gelfand
Chi Ma
Emma Prieto
Kelly D. Stone
Joshua D. Milner
Natsuko Yamakawa
Jeffrey R. Stinson
Silvia Danielian
Jonathan Zaiat
Paul R. Reynolds
Andrew L. Snow
Swadhinya Arjunaraja
Yu Zhang
Elisa Ruffo
Salomé Glauzy
Sergio D. Rosenzweig
Jennifer Stoddard
Marcelo A. Martí
Matías Oleastro
Nina Jones
Neil Romberg
Alejandro Palma
Kelsey Voss
Celeste G Nelson
Joshua J McElwee
Julie E. Niemela
Helen F. Matthews
Yuan Zhang
Andrea Bernasconi
Source :
Nature Genetics. 49:1192-1201
Publication Year :
2017
Publisher :
Springer Science and Business Media LLC, 2017.

Abstract

Few monogenic causes for severe manifestations of common allergic diseases have been identified. Through next-generation sequencing on a cohort of patients with severe atopic dermatitis with and without comorbid infections, we found eight individuals, from four families, with novel heterozygous mutations in CARD11, which encodes a scaffolding protein involved in lymphocyte receptor signaling. Disease improved over time in most patients. Transfection of mutant CARD11 expression constructs into T cell lines demonstrated both loss-of-function and dominant-interfering activity upon antigen receptor-induced activation of nuclear factor-κB and mammalian target of rapamycin complex 1 (mTORC1). Patient T cells had similar defects, as well as low production of the cytokine interferon-γ (IFN-γ). The mTORC1 and IFN-γ production defects were partially rescued by supplementation with glutamine, which requires CARD11 for import into T cells. Our findings indicate that a single hypomorphic mutation in CARD11 can cause potentially correctable cellular defects that lead to atopic dermatitis.

Details

ISSN :
15461718 and 10614036
Volume :
49
Database :
OpenAIRE
Journal :
Nature Genetics
Accession number :
edsair.doi.dedup.....0771c386e540f21e44863d09e0748ebd
Full Text :
https://doi.org/10.1038/ng.3898