Complete lung agenesis caused by complex genomic rearrangements with neo-TAD formation at the SHH locus.


Journal

Human genetics
ISSN: 1432-1203
Titre abrégé: Hum Genet
Pays: Germany
ID NLM: 7613873

Informations de publication

Date de publication:
Oct 2021
Historique:
received: 03 06 2021
accepted: 10 08 2021
pubmed: 27 8 2021
medline: 1 10 2021
entrez: 26 8 2021
Statut: ppublish

Résumé

During human organogenesis, lung development is a timely and tightly regulated developmental process under the control of a large number of signaling molecules. Understanding how genetic variants can disturb normal lung development causing different lung malformations is a major goal for dissecting molecular mechanisms during embryogenesis. Here, through exome sequencing (ES), array CGH, genome sequencing (GS) and Hi-C, we aimed at elucidating the molecular basis of bilateral isolated lung agenesis in three fetuses born to a non-consanguineous family. We detected a complex genomic rearrangement containing duplicated, triplicated and deleted fragments involving the SHH locus in fetuses presenting complete agenesis of both lungs and near-complete agenesis of the trachea, diagnosed by ultrasound screening and confirmed at autopsy following termination. The rearrangement did not include SHH itself, but several regulatory elements for lung development, such as MACS1, a major SHH lung enhancer, and the neighboring genes MNX1 and NOM1. The rearrangement incorporated parts of two topologically associating domains (TADs) including their boundaries. Hi-C of cells from one of the affected fetuses showed the formation of two novel TADs each containing SHH enhancers and the MNX1 and NOM1 genes. Hi-C together with GS indicate that the new 3D conformation is likely causative for this condition by an inappropriate activation of MNX1 included in the neo-TADs by MACS1 enhancer, further highlighting the importance of the 3D chromatin conformation in human disease.

Identifiants

pubmed: 34436670
doi: 10.1007/s00439-021-02344-6
pii: 10.1007/s00439-021-02344-6
pmc: PMC8460539
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

1459-1469

Subventions

Organisme : Deutsche Forschungsgemeinschaft
ID : SP1532/3-1
Organisme : Deutsche Forschungsgemeinschaft
ID : SP1532/5-1
Organisme : Deutsche Forschungsgemeinschaft
ID : MU 880/16-1
Organisme : Deutsche Forschungsgemeinschaft
ID : KU 1240/10-1

Informations de copyright

© 2021. The Author(s).

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Auteurs

Uirá Souto Melo (US)

Max Planck Institute for Molecular Genetics, RG Development and Disease, Berlin, Germany.
Institute of Medical Genetics and Human Genetics, Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, Germany.

Juliette Piard (J)

Centre de Génétique Humaine, Université de Franche-Comté, Besançon, France.

Björn Fischer-Zirnsak (B)

Institute of Medical Genetics and Human Genetics, Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, Germany.

Marius-Konstantin Klever (MK)

Max Planck Institute for Molecular Genetics, RG Development and Disease, Berlin, Germany.
Institute of Medical Genetics and Human Genetics, Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, Germany.

Robert Schöpflin (R)

Max Planck Institute for Molecular Genetics, RG Development and Disease, Berlin, Germany.

Martin Atta Mensah (MA)

Institute of Medical Genetics and Human Genetics, Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, Germany.
Berlin Institute of Health at Charité-Universitätsmedizin Berlin, Berlin, Germany.

Manuel Holtgrewe (M)

Berlin Institute of Health at Charité-Universitätsmedizin Berlin, Berlin, Germany.

Francine Arbez-Gindre (F)

Departments of Obstetrics and Gynecology, Pathology, and Biology, University Hospital, University of Franche-Comte, Besançon, France.

Alain Martin (A)

Departments of Obstetrics and Gynecology, Pathology, and Biology, University Hospital, University of Franche-Comte, Besançon, France.

Virginie Guigue (V)

Departments of Obstetrics and Gynecology, Pathology, and Biology, University Hospital, University of Franche-Comte, Besançon, France.
Department of Obstetrics and Gynecology, University Hospital Grenoble-Alpes, Grenoble, France.

Dominique Gaillard (D)

Department of Obstetrics and Gynecology, University Hospital Grenoble-Alpes, Grenoble, France.

Emilie Landais (E)

Department of Obstetrics and Gynecology, University Hospital Grenoble-Alpes, Grenoble, France.

Virginie Roze (V)

Departments of Obstetrics and Gynecology, Pathology, and Biology, University Hospital, University of Franche-Comte, Besançon, France.

Valerie Kremer (V)

Department of Medical Genetics, University Hospital, University Champagne-Ardennes, Reims, France.
Laboratory of Cytogenetics, University Hospital, University of Strasbourg, Strasbourg, France.
Department of Medical Genetics, University Hospital, Geneva, Switzerland.

Rajeev Ramanah (R)

Departments of Obstetrics and Gynecology, Pathology, and Biology, University Hospital, University of Franche-Comte, Besançon, France.

Christelle Cabrol (C)

Centre de Génétique Humaine, Université de Franche-Comté, Besançon, France.

Frederike L Harms (FL)

Institute of Human Genetics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.

Uwe Kornak (U)

Institute of Human Genetics, Universitätsmedizin Göttingen, Göttingen, Germany.

Malte Spielmann (M)

Max Planck Institute for Molecular Genetics, RG Development and Disease, Berlin, Germany.
Institute of Human Genetics, University of Lübeck, Lübeck, Germany.

Stefan Mundlos (S)

Max Planck Institute for Molecular Genetics, RG Development and Disease, Berlin, Germany. stefan.mundlos@charite.de.
Institute of Medical Genetics and Human Genetics, Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, Germany. stefan.mundlos@charite.de.

Lionel Van Maldergem (L)

Centre de Génétique Humaine, Université de Franche-Comté, Besançon, France.
Berlin Institute of Health at Charité-Universitätsmedizin Berlin, Berlin, Germany.
Center of Clinical Investigation (CIC), National Institute of Health and Medical Research (INSERM), CHU, Besançon, France.

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Classifications MeSH