Genome-wide identification of disease-causing copy number variations in 450 individuals with anorectal malformations.


Journal

European journal of human genetics : EJHG
ISSN: 1476-5438
Titre abrégé: Eur J Hum Genet
Pays: England
ID NLM: 9302235

Informations de publication

Date de publication:
01 2023
Historique:
received: 18 03 2022
accepted: 06 10 2022
revised: 03 07 2022
pubmed: 3 11 2022
medline: 11 1 2023
entrez: 2 11 2022
Statut: ppublish

Résumé

Anorectal malformations (ARM) represent a spectrum of rare malformations originating from a perturbated development of the embryonic hindgut. Approximately 60% occur as a part of a defined genetic syndrome or within the spectrum of additional congenital anomalies. Rare copy number variations (CNVs) have been associated with both syndromic and non-syndromic forms. The present study represents the largest study to date to explore the contribution of CNVs to the expression of ARMs. SNP-array-based molecular karyotyping was applied in 450 individuals with ARM and 4392 healthy controls. CNVs were identified from raw intensity data using PennCNV. Overlapping CNVs between cases and controls were discarded. Remaining CNVs were filtered using a stringent filter algorithm of nine filter steps. Prioritized CNVs were confirmed using qPCR. Filtering prioritized and qPCR confirmed four microscopic chromosomal anomalies and nine submicroscopic CNVs comprising seven microdeletions (del2p13.2, del4p16.2, del7q31.33, del9p24.1, del16q12.1, del18q32, del22q11.21) and two microduplications (dup2p13.2, dup17q12) in 14 individuals (12 singletons and one affected sib-pair). Within these CNVs, based on their embryonic expression data and function, we suggest FOXK2, LPP, and SALL3 as putative candidate genes. Overall, our CNV analysis identified putative microscopic and submicroscopic chromosomal rearrangements in 3% of cases. Functional characterization and re-sequencing of suggested candidate genes is warranted.

Identifiants

pubmed: 36319675
doi: 10.1038/s41431-022-01216-5
pii: 10.1038/s41431-022-01216-5
pmc: PMC9822900
doi:

Types de publication

Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

105-111

Informations de copyright

© 2022. The Author(s).

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Auteurs

Julia Fabian (J)

Institute of Human Genetics, Medical Faculty of the University Bonn & University Hospital Bonn, Bonn, Germany.

Gabriel C Dworschak (GC)

Institute of Human Genetics, Medical Faculty of the University Bonn & University Hospital Bonn, Bonn, Germany.
Department of Neuropediatrics, University Hospital Bonn, Bonn, Germany.
Institute of Anatomy, Medical Faculty, University of Bonn, Bonn, Germany.

Lea Waffenschmidt (L)

Institute of Human Genetics, Medical Faculty of the University Bonn & University Hospital Bonn, Bonn, Germany.

Luca Schierbaum (L)

Institute of Human Genetics, Medical Faculty of the University Bonn & University Hospital Bonn, Bonn, Germany.

Charlotte Bendixen (C)

Institute of Human Genetics, Medical Faculty of the University Bonn & University Hospital Bonn, Bonn, Germany.
Unit of Pediatric Surgery, Department of General, Visceral, Vascular and Thoracic Surgery, University Hospital Bonn, Bonn, Germany.

Stefanie Heilmann-Heimbach (S)

Institute of Human Genetics, Medical Faculty of the University Bonn & University Hospital Bonn, Bonn, Germany.

Sugirthan Sivalingam (S)

Institute for Medical Biometry, Informatics and Epidemiology, Medical Faculty, University of Bonn, Bonn, Germany.
Institute for Genomic Statistics and Bioinformatics, Medical Faculty, University of Bonn, Bonn, Germany.
Core Unit for Bioinformatics Data Analysis, Medical Faculty, University of Bonn, Bonn, Germany.

Andreas Buness (A)

Institute for Medical Biometry, Informatics and Epidemiology, Medical Faculty, University of Bonn, Bonn, Germany.
Institute for Genomic Statistics and Bioinformatics, Medical Faculty, University of Bonn, Bonn, Germany.
Core Unit for Bioinformatics Data Analysis, Medical Faculty, University of Bonn, Bonn, Germany.

Nicole Schwarzer (N)

SoMA, The German Patient Support Organization for Anorectal Malformations and Hirschsprung Disease, Munich, Germany.

Thomas M Boemers (TM)

Department of Pediatric Surgery and Pediatric Urology, Children's Hospital of Cologne Amsterdamer Strasse, Cologne, Germany.

Eberhard Schmiedeke (E)

Clinic for Pediatric Surgery and Pediatric Urology, Klinikum Bremen Mitte, Bremen, Germany.

Jörg Neser (J)

Department of Pediatric Surgery, General Hospital, Chemnitz, Germany.

Johannes Leonhardt (J)

Department of Pediatric Surgery, Children's Hospital Braunschweig, Braunschweig, Germany.

Ferdinand Kosch (F)

Department of Pediatric Surgery, Städtisches Klinikum Karlsruhe, Karlsruhe, Germany.

Sandra Weih (S)

Department of Pediatric Surgery, Medical Center, Faculty of Medicine, University of Freiburg, Freiburg, Germany.

Helen Maya Gielen (HM)

Department of Pediatric Surgery, Asklepios Klinik Nord Heidberg, Hamburg, Deutschland.

Stuart Hosie (S)

Muenchen Klinik gGmbH, Muenchen, Klinik Schwabing, Technische Universitaet Muenchen, Munich, Germany.

Carmen Kabs (C)

Muenchen Klinik gGmbH, Muenchen, Klinik Schwabing, Technische Universitaet Muenchen, Munich, Germany.

Markus Palta (M)

Department of Pediatric Surgery, Evangelisches Krankenhaus Hamm, Hamm, Germany.

Stefanie Märzheuser (S)

Department of Pediatric Surgery, Rostock University Medical Center, Rostock, Germany.

Lena Marie Bode (LM)

Department of Pediatric Surgery, University of Leipzig, Leipzig, Germany.

Martin Lacher (M)

Department of Pediatric Surgery, University of Leipzig, Leipzig, Germany.

Frank-Mattias Schäfer (FM)

Department of Pediatric Surgery and Pediatric Urology, Cnopfsche Kinderklinik-Klinik Hallerwiese, Nürnberg, Germany.

Maximilian Stehr (M)

Department of Pediatric Surgery and Pediatric Urology, Cnopfsche Kinderklinik-Klinik Hallerwiese, Nürnberg, Germany.

Christian Knorr (C)

Department of Pediatric Surgery and Orthopedics, University Children's Hospital Regensburg (KUNO) at the Hospital St. Hedwig of the Order of St. John, Regensburg, Germany.

Benno Ure (B)

Center of Pediatric Surgery Hannover, Hannover Medical School, Hannover, Germany.

Katharina Kleine (K)

Department of Pediatric Surgery, Evangelisches Krankenhaus Oberhausen, Oberhausen, Germany.

Udo Rolle (U)

Department of Pediatric Surgery and Pediatric Urology, Goethe University Frankfurt, Frankfurt, Germany.

Marcin Zaniew (M)

Department of Pediatrics, University of Zielona Góra, Zielona Góra, Poland.

Grote Phillip (G)

Institute of Cardiovascular Regeneration, Center for Molecular Medicine, University of Frankfurt, Frankfurt am Main, Germany.

Nadine Zwink (N)

Department of Child and Adolescent Psychiatry, University Medical Center of the Johannes Gutenberg University Mainz, Mainz, Germany.

Ekkehart Jenetzky (E)

Department of Child and Adolescent Psychiatry, University Medical Center of the Johannes Gutenberg University Mainz, Mainz, Germany.
Faculty of Health, School of Medicine, University of Witten/Herdecke, Witten, Germany.

Heiko Reutter (H)

Institute of Human Genetics, Medical Faculty of the University Bonn & University Hospital Bonn, Bonn, Germany. heiko.reutter@uk-erlangen.de.
Division of Neonatology and Pediatric Intensive Care, Department of Pediatrics and Adolescent Medicine, Friedrich-Alexander University Nürnberg-Erlangen, Erlangen, Germany. heiko.reutter@uk-erlangen.de.

Alina C Hilger (AC)

Institute of Human Genetics, Medical Faculty of the University Bonn & University Hospital Bonn, Bonn, Germany. alina.hilger@uk-erlangen.de.
Department of Pediatrics and Adolescent Medicine, Friedrich-Alexander University Nürnberg-Erlangen, Erlangen, Germany. alina.hilger@uk-erlangen.de.
Research Center On Rare Kidney Diseases (RECORD), University Hospital Erlangen, 91054, Erlangen, Germany. alina.hilger@uk-erlangen.de.

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