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BACH2 immunodeficiency illustrates an association between super-enhancers and haploinsufficiency.

Accepted version
Peer-reviewed

Type

Article

Change log

Authors

Afzali, Behdad 
Grönholm, Juha 
Vandrovcova, Jana 
O'Brien, Charlotte 
Sun, Hong-Wei 

Abstract

The transcriptional programs that guide lymphocyte differentiation depend on the precise expression and timing of transcription factors (TFs). The TF BACH2 is essential for T and B lymphocytes and is associated with an archetypal super-enhancer (SE). Single-nucleotide variants in the BACH2 locus are associated with several autoimmune diseases, but BACH2 mutations that cause Mendelian monogenic primary immunodeficiency have not previously been identified. Here we describe a syndrome of BACH2-related immunodeficiency and autoimmunity (BRIDA) that results from BACH2 haploinsufficiency. Affected subjects had lymphocyte-maturation defects that caused immunoglobulin deficiency and intestinal inflammation. The mutations disrupted protein stability by interfering with homodimerization or by causing aggregation. We observed analogous lymphocyte defects in Bach2-heterozygous mice. More generally, we observed that genes that cause monogenic haploinsufficient diseases were substantially enriched for TFs and SE architecture. These findings reveal a previously unrecognized feature of SE architecture in Mendelian diseases of immunity: heterozygous mutations in SE-regulated genes identified by whole-exome/genome sequencing may have greater significance than previously recognized.

Description

Keywords

Adrenal Cortex Hormones, Adult, Autoimmune Diseases, Basic-Leucine Zipper Transcription Factors, Colitis, Female, Fever, Haploinsufficiency, Heterozygote, Humans, Immunologic Deficiency Syndromes, Lymphopenia, Male, Middle Aged, Mutation, Pancytopenia, Pedigree, Polymorphism, Single Nucleotide, Recurrence, Respiratory Tract Infections, Splenomegaly, Syndrome, Tomography, X-Ray Computed, Young Adult

Journal Title

Nat Immunol

Conference Name

Journal ISSN

1529-2908
1529-2916

Volume Title

18

Publisher

Springer Science and Business Media LLC

Rights

All rights reserved
Sponsorship
Wellcome Trust (105663/Z/14/Z)