Whole-genome sequencing of a sporadic primary immunodeficiency cohort
File(s)PID_Nature_accepted_merged.pdf (9.71 MB)
Accepted version
Author(s)
Type
Journal Article
Abstract
Primary immunodeficiency (PID) is characterized by recurrent and often life-threatening infections, autoimmunity and cancer, and it poses major diagnostic and therapeutic challenges. Although the most severe forms of PID are identified in early childhood, most patients present in adulthood, typically with no apparent family history and a variable clinical phenotype of widespread immune dysregulation: about 25% of patients have autoimmune disease, allergy is prevalent and up to 10% develop lymphoid malignancies1-3. Consequently, in sporadic (or non-familial) PID genetic diagnosis is difficult and the role of genetics is not well defined. Here we address these challenges by performing whole-genome sequencing in a large PID cohort of 1,318 participants. An analysis of the coding regions of the genome in 886 index cases of PID found that disease-causing mutations in known genes that are implicated in monogenic PID occurred in 10.3% of these patients, and a Bayesian approach (BeviMed4) identified multiple new candidate PID-associated genes, including IVNS1ABP. We also examined the noncoding genome, and found deletions in regulatory regions that contribute to disease causation. In addition, we used a genome-wide association study to identify loci that are associated with PID, and found evidence for the colocalization of-and interplay between-novel high-penetrance monogenic variants and common variants (at the PTPN2 and SOCS1 loci). This begins to explain the contribution of common variants to the variable penetrance and phenotypic complexity that are observed in PID. Thus, using a cohort-based whole-genome-sequencing approach in the diagnosis of PID can increase diagnostic yield and further our understanding of the key pathways that influence immune responsiveness in humans.
Date Issued
2020-07-02
Date Acceptance
2020-02-26
Citation
Nature, 2020, 583, pp.90-95
ISSN
0028-0836
Publisher
Nature Research
Start Page
90
End Page
95
Journal / Book Title
Nature
Volume
583
Copyright Statement
© 2020 Springer Nature Limited.
Sponsor
Wellcome Trust
Identifier
https://www.ncbi.nlm.nih.gov/pubmed/32499645
PII: 10.1038/s41586-020-2265-1
Grant Number
206617/A/17/Z
Subjects
Science & Technology
Multidisciplinary Sciences
Science & Technology - Other Topics
COMMON VARIABLE IMMUNODEFICIENCY
IUIS PHENOTYPIC CLASSIFICATION
GENETIC-VARIATION
STAT1 MUTATIONS
VARIANTS
ASSOCIATION
DEFICIENCY
SUPPRESSOR
GAIN
RARE
Actin-Related Protein 2-3 Complex
Bayes Theorem
Cohort Studies
Female
Genome-Wide Association Study
Humans
Male
Primary Immunodeficiency Diseases
Protein Tyrosine Phosphatase, Non-Receptor Type 2
RNA-Binding Proteins
Regulatory Sequences, Nucleic Acid
Suppressor of Cytokine Signaling 1 Protein
Transcription Factors
Whole Genome Sequencing
Primary Immunodeficiency Consortium for the NIHR Bioresource
Humans
RNA-Binding Proteins
Transcription Factors
Bayes Theorem
Cohort Studies
Regulatory Sequences, Nucleic Acid
Female
Male
Actin-Related Protein 2-3 Complex
Protein Tyrosine Phosphatase, Non-Receptor Type 2
Genome-Wide Association Study
Suppressor of Cytokine Signaling 1 Protein
Whole Genome Sequencing
Primary Immunodeficiency Diseases
General Science & Technology
Publication Status
Published
Coverage Spatial
England
Date Publish Online
2020-05-06