HLA-DQB1*03:01 strongly affects age of onset of type 1 narcolepsy independently of DQA1 and ethnicity

L Lisan Zhang (Department of Neurology, Sir Run Run Shaw Hospital, School of Medicine, Zhejiang University) S Shuo Cai (Centre of Biomedical Systems and Informatics, Zhejiang University—University of Edinburgh Institute, Zhejiang University School of Medicine) A Ashton Teng (Department of Psychiatry and Behavioral Sciences, Center for Sleep Sciences and Medicine, Stanford University) L Ling Lin (Department of Psychiatry and Behavioral Sciences, Center for Sleep Sciences and Medicine, Stanford University) F Fang Han (Division of Sleep Medicine, The Peking University People’s Hospital) H Han Yan S Seung-Chul Hong (Department of Neuropsychiatry, College of Medicine, St. Vincent’s Hospital, Catholic University of Korea) F Fabio Pizza (Department of Biomedical and Neuromotor Sciences, University of Bologna) G Giuseppe Plazzi (Istituto di Ricovero e Cura a Carattere Scientifico - IRCCS Institute of Neurological Sciences) L Luca Morandi (Department of Biomedical and Neuromotor Sciences, University of Bologna) A Ambra Stefani (Department of Neurology, Innsbruck Medical University) B Birgit Högl (Department of Neurology, Innsbruck Medical University) M Michel Lecendreux (Pediatric Sleep Center and National Reference Center for Narcolepsy and Idiopathic Hypersomnia Hospital Robert Debre) P Patrice Bourgin (Department of Sleep Medicine, Strasbourg University Hospital, Strasbourg University) I Isabelle Arnulf (Sleep Disorder Unit, Pitié-Salpêtrière Hospital, Assistance Publique-Hopitaux de Paris) S Stine Knudsen-Heier (Norwegian Centre of Expertise for Neurodevelopment Disorders and Hypersomnias, Department of Rare Disorders, Oslo University Hospital and University of Oslo) T Takashi Kanbayashi (International Institute for Integrative Sleep Medicine (WPI-IIIS), University of Tsukuba) Y Yu-Shu Huang P Poul Jennum (Danish Center for Sleep Medicine, Department of Clinical Neurophysiology, University of Copenhagen, Glostrup Hospital) K Karel Sonka (Department of Neurology and Centre of Clinical Neurosciences, First Faculty of Medicine, Charles University and General University Hospital) S Sona Nevsimalova (Department of Neurology and Centre of Clinical Neurosciences, First Faculty of Medicine, Charles University and General University Hospital) M Makoto Honda (Department of Psychiatry and Behavioral Sciences, Tokyo Metropolitan Institute of Medical Science) F Fernando Morgadinho Coelho (Departamento de Psicobiologia, Universidade Federal de São Paulo) Z Zerrin Pelin (Faculty of Health Sciences, Hasan Kalyoncu University) L Luigi Ferini-Strambi (Sleep Disorders Center, Division of Neuroscience, Ospedale San Raffaele, Università Vita-Salute) T Taku Miyagawa (Department of Human Genetics, Graduate School of Medicine, The University of Tokyo) S Seik-Soon Khor (Department of Human Genetics, Graduate School of Medicine, The University of Tokyo) K Katsushi Tokunaga W Wanlu Liu (Centre of Biomedical Systems and Informatics, Zhejiang University—University of Edinburgh Institute, Zhejiang University School of Medicine) E Emmanuel J. Mignot (Department of Psychiatry and Behavioral Sciences, Center for Sleep Sciences and Medicine, Stanford University)

Abstract

Type 1 narcolepsy (T1N), an autoimmune disease associated with a disruption of hypocretin/orexin neurons, has conserved genetic effects transcending cultures and ethnicities. We pooled data from 5,339 cases from China, Europe, Korea, Japan, and the United States to conduct the first transethnic genome-wide association study (GWAS) on age of onset. Only one strong GWAS significant effect was observed across all ethnicities, summarized by the presence of human leukocyte antigen (HLA)-DQB1*03:01, and centered around the coding region of this gene. In contrast, HLA-DQB1*06:02-positive heterodimer (DQ0602) dosage did not strongly affect onset, and other known narcolepsy-associated genetic loci had minor effects. The HLA-DQB1*03:01 effect (mean −3.47 y, P = 1.7 × 10 −18 ) showed no heterogeneity across ethnic groups and was independent of common allelic variation at HLA-DQA1 in cis of HLA-DQB1*03:01 (DQA1*03:03; DQA1*05:05; DQA1*06:01). This effect may be due to a peptide being presented by all DQ0301 heterodimers (which are tolerant at the P1 binding position), or it may stem from genetic effects of HLA on T cell receptor genes TCRA and TCRB usage that influence the TCR repertoire. Using bulk and single-cell RNA sequencing data across Chinese and Caucasians, who have distinct patterns of linkage disequilibrium around DQB1*03:01, we found that HLA-DQB1*03:01 alters TCR repertoire at specific positions, most significantly within the CDR2α, CDR2β, and CDR3β loops. These results illustrate the remarkable conservation of genetic effects in narcolepsy across ethnicity. The identification of the disease-causing T cells will be crucial for elucidating how this finding relates to the underlying pathophysiology.

Article Details

Volume / Issue Vol. 122, Issue 50
Published December 16, 2025
ISSN 0027-8424
Publisher National Academy of Sciences

Authors (30)

L

Lisan Zhang

Department of Neurology, Sir Run Run Shaw Hospital, School of Medicine, Zhejiang University

S

Shuo Cai

Centre of Biomedical Systems and Informatics, Zhejiang University—University of Edinburgh Institute, Zhejiang University School of Medicine

A

Ashton Teng

Department of Psychiatry and Behavioral Sciences, Center for Sleep Sciences and Medicine, Stanford University

L

Ling Lin

Department of Psychiatry and Behavioral Sciences, Center for Sleep Sciences and Medicine, Stanford University

F

Fang Han

Division of Sleep Medicine, The Peking University People’s Hospital

H

Han Yan

S

Seung-Chul Hong

Department of Neuropsychiatry, College of Medicine, St. Vincent’s Hospital, Catholic University of Korea

F

Fabio Pizza

Department of Biomedical and Neuromotor Sciences, University of Bologna

G

Giuseppe Plazzi

Istituto di Ricovero e Cura a Carattere Scientifico - IRCCS Institute of Neurological Sciences

L

Luca Morandi

Department of Biomedical and Neuromotor Sciences, University of Bologna

A

Ambra Stefani

Department of Neurology, Innsbruck Medical University

B

Birgit Högl

Department of Neurology, Innsbruck Medical University

M

Michel Lecendreux

Pediatric Sleep Center and National Reference Center for Narcolepsy and Idiopathic Hypersomnia Hospital Robert Debre

P

Patrice Bourgin

Department of Sleep Medicine, Strasbourg University Hospital, Strasbourg University

I

Isabelle Arnulf

Sleep Disorder Unit, Pitié-Salpêtrière Hospital, Assistance Publique-Hopitaux de Paris

S

Stine Knudsen-Heier

Norwegian Centre of Expertise for Neurodevelopment Disorders and Hypersomnias, Department of Rare Disorders, Oslo University Hospital and University of Oslo

T

Takashi Kanbayashi

International Institute for Integrative Sleep Medicine (WPI-IIIS), University of Tsukuba

Y

Yu-Shu Huang

P

Poul Jennum

Danish Center for Sleep Medicine, Department of Clinical Neurophysiology, University of Copenhagen, Glostrup Hospital

K

Karel Sonka

Department of Neurology and Centre of Clinical Neurosciences, First Faculty of Medicine, Charles University and General University Hospital

S

Sona Nevsimalova

Department of Neurology and Centre of Clinical Neurosciences, First Faculty of Medicine, Charles University and General University Hospital

M

Makoto Honda

Department of Psychiatry and Behavioral Sciences, Tokyo Metropolitan Institute of Medical Science

F

Fernando Morgadinho Coelho

Departamento de Psicobiologia, Universidade Federal de São Paulo

Z

Zerrin Pelin

Faculty of Health Sciences, Hasan Kalyoncu University

L

Luigi Ferini-Strambi

Sleep Disorders Center, Division of Neuroscience, Ospedale San Raffaele, Università Vita-Salute

T

Taku Miyagawa

Department of Human Genetics, Graduate School of Medicine, The University of Tokyo

S

Seik-Soon Khor

Department of Human Genetics, Graduate School of Medicine, The University of Tokyo

K

Katsushi Tokunaga

W

Wanlu Liu

Centre of Biomedical Systems and Informatics, Zhejiang University—University of Edinburgh Institute, Zhejiang University School of Medicine

E

Emmanuel J. Mignot

Department of Psychiatry and Behavioral Sciences, Center for Sleep Sciences and Medicine, Stanford University