Coding and noncoding variants in EBF3 are involved in HADDS and simplex autism.


Journal

Human genomics
ISSN: 1479-7364
Titre abrégé: Hum Genomics
Pays: England
ID NLM: 101202210

Informations de publication

Date de publication:
13 07 2021
Historique:
received: 03 05 2021
accepted: 17 06 2021
entrez: 14 7 2021
pubmed: 15 7 2021
medline: 5 2 2022
Statut: epublish

Résumé

Previous research in autism and other neurodevelopmental disorders (NDDs) has indicated an important contribution of protein-coding (coding) de novo variants (DNVs) within specific genes. The role of de novo noncoding variation has been observable as a general increase in genetic burden but has yet to be resolved to individual functional elements. In this study, we assessed whole-genome sequencing data in 2671 families with autism (discovery cohort of 516 families, replication cohort of 2155 families). We focused on DNVs in enhancers with characterized in vivo activity in the brain and identified an excess of DNVs in an enhancer named hs737. We adapted the fitDNM statistical model to work in noncoding regions and tested enhancers for excess of DNVs in families with autism. We found only one enhancer (hs737) with nominal significance in the discovery (p = 0.0172), replication (p = 2.5 × 10 In this study, we identify DNVs in the hs737 enhancer in individuals with autism. Through multiple approaches, we find hs737 targets the gene EBF3 that is genome-wide significant in NDDs. By assessment of noncoding variation and the genes they affect, we are beginning to understand their impact on gene regulatory networks in NDDs.

Sections du résumé

BACKGROUND
Previous research in autism and other neurodevelopmental disorders (NDDs) has indicated an important contribution of protein-coding (coding) de novo variants (DNVs) within specific genes. The role of de novo noncoding variation has been observable as a general increase in genetic burden but has yet to be resolved to individual functional elements. In this study, we assessed whole-genome sequencing data in 2671 families with autism (discovery cohort of 516 families, replication cohort of 2155 families). We focused on DNVs in enhancers with characterized in vivo activity in the brain and identified an excess of DNVs in an enhancer named hs737.
RESULTS
We adapted the fitDNM statistical model to work in noncoding regions and tested enhancers for excess of DNVs in families with autism. We found only one enhancer (hs737) with nominal significance in the discovery (p = 0.0172), replication (p = 2.5 × 10
CONCLUSIONS
In this study, we identify DNVs in the hs737 enhancer in individuals with autism. Through multiple approaches, we find hs737 targets the gene EBF3 that is genome-wide significant in NDDs. By assessment of noncoding variation and the genes they affect, we are beginning to understand their impact on gene regulatory networks in NDDs.

Identifiants

pubmed: 34256850
doi: 10.1186/s40246-021-00342-3
pii: 10.1186/s40246-021-00342-3
pmc: PMC8278787
doi:

Substances chimiques

EBF3 protein, human 0
Transcription Factors 0

Types de publication

Journal Article Research Support, N.I.H., Extramural Research Support, U.S. Gov't, Non-P.H.S.

Langues

eng

Sous-ensembles de citation

IM

Pagination

44

Subventions

Organisme : NHGRI NIH HHS
ID : R01 HG003988
Pays : United States
Organisme : NHGRI NIH HHS
ID : UM1 HG008901
Pays : United States
Organisme : NHGRI NIH HHS
ID : K01 HG010498
Pays : United States
Organisme : NHGRI NIH HHS
ID : U24 HG008956
Pays : United States
Organisme : NIGMS NIH HHS
ID : T32 GM139774
Pays : United States
Organisme : NIMH NIH HHS
ID : R00 MH117165
Pays : United States

Informations de copyright

© 2021. The Author(s).

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Auteurs

Evin M Padhi (EM)

Department of Genetics, Washington University School of Medicine, 4523 Clayton Avenue, Campus Box 8232, St. Louis, MO, 63110, USA.

Tristan J Hayeck (TJ)

Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, 19104, USA.
Department of Pathology and Laboratory Medicine, Children's Hospital of Philadelphia, Philadelphia, PA, 19104, USA.

Zhang Cheng (Z)

Center for Epigenomics, University of California San Diego School of Medicine, 9500 Gilman Drive, La Jolla, CA, 92093, USA.

Sumantra Chatterjee (S)

Center for Human Genetics and Genomics, NYU School of Medicine, New York, NY, 10016, USA.

Brandon J Mannion (BJ)

Environmental Genomics and Systems Biology Division, Lawrence Berkeley National Laboratory, Berkeley, CA, 94720, USA.

Marta Byrska-Bishop (M)

New York Genome Center, New York, NY, 10013, USA.

Marjolaine Willems (M)

University of Montpellier, département de Génétique, maladies rares médecine personnalisée, U 1298, CHU Montpellier, University of Montpellier, Montpellier, France.

Lucile Pinson (L)

University of Montpellier, département de Génétique, maladies rares médecine personnalisée, U 1298, CHU Montpellier, University of Montpellier, Montpellier, France.

Sylvia Redon (S)

CHU Brest, Inserm, Univ Brest, EFS,UMR 1078, GGB, F-29200, Brest, France.

Caroline Benech (C)

CHU Brest, Inserm, Univ Brest, EFS,UMR 1078, GGB, F-29200, Brest, France.

Kevin Uguen (K)

CHU Brest, Inserm, Univ Brest, EFS,UMR 1078, GGB, F-29200, Brest, France.

Séverine Audebert-Bellanger (S)

Service de Génétique Médicale, CHRU de Brest, Brest, France.

Cédric Le Marechal (C)

CHU Brest, Inserm, Univ Brest, EFS,UMR 1078, GGB, F-29200, Brest, France.

Claude Férec (C)

CHU Brest, Inserm, Univ Brest, EFS,UMR 1078, GGB, F-29200, Brest, France.

Stephanie Efthymiou (S)

Department of Neuromuscular Disorders, UCL Institute of Neurology, Queen Square, London, WC1N 3BG, UK.

Fatima Rahman (F)

Development and Behavioral Pediatrics Department, Institute of Child Health and Children Hospital, Lahore, Pakistan.

Shazia Maqbool (S)

Department of Neuromuscular Disorders, UCL Institute of Neurology, Queen Square, London, WC1N 3BG, UK.
Development and Behavioral Pediatrics Department, Institute of Child Health and Children Hospital, Lahore, Pakistan.

Reza Maroofian (R)

Department of Neuromuscular Disorders, UCL Institute of Neurology, Queen Square, London, WC1N 3BG, UK.

Henry Houlden (H)

Department of Neuromuscular Disorders, UCL Institute of Neurology, Queen Square, London, WC1N 3BG, UK.

Rajeeva Musunuri (R)

New York Genome Center, New York, NY, 10013, USA.

Giuseppe Narzisi (G)

New York Genome Center, New York, NY, 10013, USA.

Avinash Abhyankar (A)

New York Genome Center, New York, NY, 10013, USA.

Riana D Hunter (RD)

Environmental Genomics and Systems Biology Division, Lawrence Berkeley National Laboratory, Berkeley, CA, 94720, USA.

Jennifer Akiyama (J)

Environmental Genomics and Systems Biology Division, Lawrence Berkeley National Laboratory, Berkeley, CA, 94720, USA.

Lauren E Fries (LE)

Center for Human Genetics and Genomics, NYU School of Medicine, New York, NY, 10016, USA.

Jeffrey K Ng (JK)

Department of Genetics, Washington University School of Medicine, 4523 Clayton Avenue, Campus Box 8232, St. Louis, MO, 63110, USA.

Elvisa Mehinovic (E)

Department of Genetics, Washington University School of Medicine, 4523 Clayton Avenue, Campus Box 8232, St. Louis, MO, 63110, USA.

Nick Stong (N)

Institute for Genomic Medicine, Columbia University, New York, NY, 10027, USA.

Andrew S Allen (AS)

Center for Statistical Genetics and Genomics, Duke University, Durham, NC, 27708, USA.
Division of Integrative Genomics, Duke University, Durham, NC, 27708, USA.
Department of Biostatistics and Bioinformatics, Duke University, Durham, NC, 27708, USA.

Diane E Dickel (DE)

Environmental Genomics and Systems Biology Division, Lawrence Berkeley National Laboratory, Berkeley, CA, 94720, USA.

Raphael A Bernier (RA)

Department of Psychiatry and Behavioral Sciences, University of Washington, Seattle, WA, 98195, USA.

David U Gorkin (DU)

Center for Epigenomics, University of California San Diego School of Medicine, 9500 Gilman Drive, La Jolla, CA, 92093, USA.
Department of Biology, Emory University, Atlanta, GA, 30322, USA.

Len A Pennacchio (LA)

Environmental Genomics and Systems Biology Division, Lawrence Berkeley National Laboratory, Berkeley, CA, 94720, USA.
U.S. Department of Energy Joint Genome Institute, Walnut Creek, CA, 94598, USA.

Michael C Zody (MC)

New York Genome Center, New York, NY, 10013, USA.

Tychele N Turner (TN)

Department of Genetics, Washington University School of Medicine, 4523 Clayton Avenue, Campus Box 8232, St. Louis, MO, 63110, USA. tychele@wustl.edu.

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