Whole-genome sequencing of patients with rare diseases in a national health system
Author(s)
Type
Journal Article
Abstract
Most patients with rare diseases do not receive a molecular diagnosis and the aetiological variants and causative genes for more than half such disorders remain to be discovered1. Here we used whole-genome sequencing (WGS) in a national health system to streamline diagnosis and to discover unknown aetiological variants in the coding and non-coding regions of the genome. We generated WGS data for 13,037 participants, of whom 9,802 had a rare disease, and provided a genetic diagnosis to 1,138 of the 7,065 extensively phenotyped participants. We identified 95 Mendelian associations between genes and rare diseases, of which 11 have been discovered since 2015 and at least 79 are confirmed to be aetiological. By generating WGS data of UK Biobank participants2, we found that rare alleles can explain the presence of some individuals in the tails of a quantitative trait for red blood cells. Finally, we identified four novel non-coding variants that cause disease through the disruption of transcription of ARPC1B, GATA1, LRBA and MPL. Our study demonstrates a synergy by using WGS for diagnosis and aetiological discovery in routine healthcare.
Date Issued
2020-07-02
Date Acceptance
2020-05-05
Citation
Nature, 2020, 583, pp.96-102
ISSN
0028-0836
Publisher
Nature Research
Start Page
96
End Page
102
Journal / Book Title
Nature
Volume
583
Copyright Statement
© The Author(s), under exclusive licence to Springer Nature Limited 2020
Sponsor
John Wyeth & Brother Limited
Medical Research Council (MRC)
Wellcome Trust
Wellcome Trust
Identifier
http://gateway.webofknowledge.com/gateway/Gateway.cgi?GWVersion=2&SrcApp=PARTNER_APP&SrcAuth=LinksAMR&KeyUT=WOS:000556405300001&DestLinkType=FullRecord&DestApp=ALL_WOS&UsrCustomerID=1ba7043ffcc86c417c072aa74d649202
Grant Number
N/A
MR/J011711/1
206617/A/17/Z
107469/Z/15/Z
Subjects
Science & Technology
Multidisciplinary Sciences
Science & Technology - Other Topics
MUTATIONS
VARIANTS
THROMBOCYTOPENIA
MACROTHROMBOCYTOPENIA
ASSOCIATION
DIAGNOSIS
LINKS
Publication Status
Published
Date Publish Online
2020-06-24