Paralog Studies Augment Gene Discovery: DDX and DHX Genes.


Journal

American journal of human genetics
ISSN: 1537-6605
Titre abrégé: Am J Hum Genet
Pays: United States
ID NLM: 0370475

Informations de publication

Date de publication:
01 08 2019
Historique:
received: 10 12 2018
accepted: 31 05 2019
pubmed: 2 7 2019
medline: 17 3 2020
entrez: 2 7 2019
Statut: ppublish

Résumé

Members of a paralogous gene family in which variation in one gene is known to cause disease are eight times more likely to also be associated with human disease. Recent studies have elucidated DHX30 and DDX3X as genes for which pathogenic variant alleles are involved in neurodevelopmental disorders. We hypothesized that variants in paralogous genes encoding members of the DExD/H-box RNA helicase superfamily might also underlie developmental delay and/or intellectual disability (DD and/or ID) disease phenotypes. Here we describe 15 unrelated individuals who have DD and/or ID, central nervous system (CNS) dysfunction, vertebral anomalies, and dysmorphic features and were found to have probably damaging variants in DExD/H-box RNA helicase genes. In addition, these individuals exhibit a variety of other tissue and organ system involvement including ocular, outer ear, hearing, cardiac, and kidney tissues. Five individuals with homozygous (one), compound-heterozygous (two), or de novo (two) missense variants in DHX37 were identified by exome sequencing. We identified ten total individuals with missense variants in three other DDX/DHX paralogs: DHX16 (four individuals), DDX54 (three individuals), and DHX34 (three individuals). Most identified variants are rare, predicted to be damaging, and occur at conserved amino acid residues. Taken together, these 15 individuals implicate the DExD/H-box helicases in both dominantly and recessively inherited neurodevelopmental phenotypes and highlight the potential for more than one disease mechanism underlying these disorders.

Identifiants

pubmed: 31256877
pii: S0002-9297(19)30226-5
doi: 10.1016/j.ajhg.2019.06.001
pmc: PMC6698803
pii:
doi:

Substances chimiques

Neoplasm Proteins 0
DDX54 protein, human EC 2.7.7.-
DHX16 protein, human EC 2.7.7.-
DHX34 protein, human EC 2.7.7.-
DEAD-box RNA Helicases EC 3.6.4.13
RNA Helicases EC 3.6.4.13

Types de publication

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

Langues

eng

Sous-ensembles de citation

IM

Pagination

302-316

Subventions

Organisme : NHGRI NIH HHS
ID : UM1 HG006542
Pays : United States
Organisme : NINDS NIH HHS
ID : R35 NS105078
Pays : United States
Organisme : NHGRI NIH HHS
ID : K08 HG008986
Pays : United States
Organisme : NIDDK NIH HHS
ID : R01 DK078121
Pays : United States
Organisme : NIDDK NIH HHS
ID : K12 DK083014
Pays : United States
Organisme : NHGRI NIH HHS
ID : UM1 HG006493
Pays : United States
Organisme : NIGMS NIH HHS
ID : T32 GM008307
Pays : United States

Informations de copyright

Copyright © 2019 American Society of Human Genetics. Published by Elsevier Inc. All rights reserved.

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Auteurs

Ingrid Paine (I)

Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX 77030, USA.

Jennifer E Posey (JE)

Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX 77030, USA.

Christopher M Grochowski (CM)

Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX 77030, USA.

Shalini N Jhangiani (SN)

Human Genome Sequencing Center, Baylor College of Medicine, Houston, TX 77030, USA.

Sarah Rosenheck (S)

Stanley Institute for Cognitive Genomics, Cold Spring Harbor Laboratory, NY 11724, USA.

Robert Kleyner (R)

Stanley Institute for Cognitive Genomics, Cold Spring Harbor Laboratory, NY 11724, USA.

Taylor Marmorale (T)

Stanley Institute for Cognitive Genomics, Cold Spring Harbor Laboratory, NY 11724, USA.

Margaret Yoon (M)

Stanley Institute for Cognitive Genomics, Cold Spring Harbor Laboratory, NY 11724, USA.

Kai Wang (K)

Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.

Reid Robison (R)

Utah Foundation for Biomedical Research, Salt Lake City, UT 84107, USA.

Gerarda Cappuccio (G)

Department of Translational Medicine, University of Naples "Federico II," 80131 Napoli, Italy; Telethon Institute of Genetics and Medicine, 80078 Pozzuoli, Italy.

Michele Pinelli (M)

Department of Translational Medicine, University of Naples "Federico II," 80131 Napoli, Italy; Telethon Institute of Genetics and Medicine, 80078 Pozzuoli, Italy.

Adriano Magli (A)

Department of Pediatric Ophthalmology, University of Salerno, 84081 Baronissi SA, Italy.

Zeynep Coban Akdemir (Z)

Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX 77030, USA.

Joannie Hui (J)

Department of Pediatrics, Prince of Wales Hospital, the Chinese University of Hong Kong, Hong Kong SAR, China.

Wai Lan Yeung (WL)

Department of Pediatrics and Adolescent Medicine, Alice Ho Miu Ling Nethersole Hospital, Hong Kong SAR, China.

Bibiana K Y Wong (BKY)

Department of Obstetrics and Gynecology, Baylor College of Medicine, Houston, TX 77030, USA; The Jan and Dan Duncan Neurological Research Institute, Texas Children's Hospital, Houston, TX 77030, USA.

Lucia Ortega (L)

Medical Genetics Department, Cook Children's Hospital, Fort Worth, TX 76104, USA.

Mir Reza Bekheirnia (MR)

Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX 77030, USA; Department of Pediatrics, Section of Pediatric Renal, Baylor College of Medicine, Houston, TX 77030, USA; Department of Genetics, Texas Children's Hospital, Houston, TX 76104, USA.

Tatjana Bierhals (T)

Institute of Human Genetics, University Medical Center Hamburg-Eppendorf, 20246 Hamburg, Germany.

Maja Hempel (M)

Institute of Human Genetics, University Medical Center Hamburg-Eppendorf, 20246 Hamburg, Germany.

Jessika Johannsen (J)

Department of Pediatrics, University Medical Center Hamburg-Eppendorf, 20246 Hamburg, Germany.

René Santer (R)

Department of Pediatrics, University Medical Center Hamburg-Eppendorf, 20246 Hamburg, Germany.

Dilek Aktas (D)

DAMAGEN Genetic Diagnostic Center, 06690 Ankara, Turkey.

Mehmet Alikasifoglu (M)

DAMAGEN Genetic Diagnostic Center, 06690 Ankara, Turkey.

Sevcan Bozdogan (S)

Department of Medical Genetics, Cukurova University Faculty of Medicine, 01330 Adana, Turkey.

Hatip Aydin (H)

Department of Medical Genetics, Medical Faculty of Namik Kemal University, Tekirdag 59100, Turkey.

Ender Karaca (E)

Department of Genetics, University of Alabama at Birmingham, Birmingham, AL 35294, USA.

Yavuz Bayram (Y)

Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York City, NY 10029, USA.

Hadas Ityel (H)

Division of Nephrology, Department of Pediatrics, Harvard Medical School, Boston, MA 02115, USA.

Michael Dorschner (M)

Center for Precision Diagnostics, University of Washington, Seattle, WA 98195, USA.

Janson J White (JJ)

Department of Pediatrics, University of Washington, Seattle, WA 98195, USA.

Ekkehard Wilichowski (E)

Department of Pediatrics and Pediatric Neurology, Georg-August-Universität Göttingen, 37075 Göttingen, Germany.

Saskia B Wortmann (SB)

Institute of Human Genetics, Technical University München, 81675 Munich, Germany; Institute of Human Genetics, Helmholtz Zentrum Munchen, 85764 Neuherberg, Germany; University Children's Hospital, Paracelsus Medical University, 5020 Salsburg, Austria.

Erasmo B Casella (EB)

Children's Institute, Hospital das Clinicas, University of Sao Paulo, 05405-000 Sao Paulo, Brazil.

Joao Paulo Kitajima (JP)

Mendelics Genomic Analysis, 04013-000 Sao Paulo, Brazil.

Fernando Kok (F)

Mendelics Genomic Analysis, 04013-000 Sao Paulo, Brazil; Department of Neurology, University of Sao Paulo School of Medicine, 01246-903 Sao Paulo, Brazil.

Fabiola Monteiro (F)

Mendelics Genomic Analysis, 04013-000 Sao Paulo, Brazil.

Donna M Muzny (DM)

Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX 77030, USA; Human Genome Sequencing Center, Baylor College of Medicine, Houston, TX 77030, USA.

Michael Bamshad (M)

Department of Pediatrics, University of Washington, Seattle, WA 98195, USA; Department of Genome Sciences, University of Washington, Seattle, WA 98195, USA; Division of Genetic Medicine, University of Washington, Seattle, WA 98195, USA.

Richard A Gibbs (RA)

Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX 77030, USA; Human Genome Sequencing Center, Baylor College of Medicine, Houston, TX 77030, USA.

V Reid Sutton (VR)

Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX 77030, USA.

Hilde Van Esch (H)

Center for Human Genetics, University Hospitals Leuven, 3000 Leuven, Belgium.

Nicola Brunetti-Pierri (N)

Department of Translational Medicine, University of Naples "Federico II," 80131 Napoli, Italy; Telethon Institute of Genetics and Medicine, 80078 Pozzuoli, Italy.

Friedhelm Hildebrandt (F)

Division of Nephrology, Department of Pediatrics, Harvard Medical School, Boston, MA 02115, USA.

Ariel Brautbar (A)

Medical Genetics Department, Cook Children's Hospital, Fort Worth, TX 76104, USA.

Ignatia B Van den Veyver (IB)

Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX 77030, USA; Department of Obstetrics and Gynecology, Baylor College of Medicine, Houston, TX 77030, USA; The Jan and Dan Duncan Neurological Research Institute, Texas Children's Hospital, Houston, TX 77030, USA.

Ian Glass (I)

Division of Genetic Medicine, University of Washington, Seattle, WA 98195, USA.

Davor Lessel (D)

Institute of Human Genetics, University Medical Center Hamburg-Eppendorf, 20246 Hamburg, Germany.

Gholson J Lyon (GJ)

Stanley Institute for Cognitive Genomics, Cold Spring Harbor Laboratory, NY 11724, USA; Utah Foundation for Biomedical Research, Salt Lake City, UT 84107, USA; Institute for Basic Research in Developmental Disabilities, Staten Island, NY 10314, USA.

James R Lupski (JR)

Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX 77030, USA; Human Genome Sequencing Center, Baylor College of Medicine, Houston, TX 77030, USA; Department of Pediatrics, Baylor College of Medicine, Houston, TX 77030, USA; Texas Children's Hospital, Houston, TX 77030, USA. Electronic address: jlupski@bcm.edu.

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