Genome sequencing in persistently unsolved white matter disorders.


Journal

Annals of clinical and translational neurology
ISSN: 2328-9503
Titre abrégé: Ann Clin Transl Neurol
Pays: United States
ID NLM: 101623278

Informations de publication

Date de publication:
01 2020
Historique:
received: 01 10 2019
revised: 05 11 2019
accepted: 05 11 2019
pubmed: 9 1 2020
medline: 2 2 2021
entrez: 9 1 2020
Statut: ppublish

Résumé

Genetic white matter disorders have heterogeneous etiologies and overlapping clinical presentations. We performed a study of the diagnostic efficacy of genome sequencing in 41 unsolved cases with prior exome sequencing, resolving an additional 14 from an historical cohort (n = 191). Reanalysis in the context of novel disease-associated genes and improved variant curation and annotation resolved 64% of cases. The remaining diagnoses were directly attributable to genome sequencing, including cases with small and large copy number variants (CNVs) and variants in deep intronic and technically difficult regions. Genome sequencing, in combination with other methodologies, achieved a diagnostic yield of 85% in this retrospective cohort.

Identifiants

pubmed: 31912665
doi: 10.1002/acn3.50957
pmc: PMC6952322
doi:

Types de publication

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

Langues

eng

Sous-ensembles de citation

IM

Pagination

144-152

Subventions

Organisme : Australian National Health and Medical Research Council
ID : 1068278
Pays : International
Organisme : CIHR
Pays : Canada

Commentaires et corrections

Type : ErratumIn

Informations de copyright

© 2020 The Authors. Annals of Clinical and Translational Neurology published by Wiley Periodicals, Inc on behalf of American Neurological Association.

Références

Genome Res. 2017 Nov;27(11):1895-1903
pubmed: 28887402
Mol Genet Metab. 2015 Apr;114(4):501-515
pubmed: 25655951
Cell. 2019 Jan 24;176(3):535-548.e24
pubmed: 30661751
J Med Genet. 2017 May;54(5):330-337
pubmed: 27799409
Genet Med. 2015 May;17(5):405-24
pubmed: 25741868
Fly (Austin). 2012 Apr-Jun;6(2):80-92
pubmed: 22728672
Acta Neuropathol. 2017 Sep;134(3):351-382
pubmed: 28638987
Brain. 2017 Oct 1;140(10):2550-2556
pubmed: 28969374
Am J Hum Genet. 2019 May 2;104(5):925-935
pubmed: 30982609
Front Mol Neurosci. 2018 Apr 04;11:104
pubmed: 29670509
Ann Neurol. 2016 Jun;79(6):1031-1037
pubmed: 27159321
Am J Med Genet A. 2018 Apr;176(4):925-935
pubmed: 29436146
Am J Hum Genet. 2015 Sep 3;97(3):457-64
pubmed: 26299366
Methods Mol Biol. 2010;653:249-57
pubmed: 20721748
Nat Genet. 2016 Oct;48(10):1185-92
pubmed: 27571260
Bioinformatics. 2016 Aug 1;32(15):2375-7
pubmed: 27153601
Genet Med. 2017 Feb;19(2):224-235
pubmed: 27513193
Clin Genet. 2011 Aug;80(2):161-6
pubmed: 21644943
Neuropediatrics. 2016 Dec;47(6):349-354
pubmed: 27564080
Curr Protoc Bioinformatics. 2013;43:11.10.1-11.10.33
pubmed: 25431634
Neurogenetics. 2017 Dec;18(4):185-194
pubmed: 28842795
J Med Genet. 2016 Feb;53(2):132-7
pubmed: 26545878
Brain. 2014 Apr;137(Pt 4):1019-29
pubmed: 24566671
Eur J Hum Genet. 2018 May;26(5):740-744
pubmed: 29453418

Auteurs

Guy Helman (G)

Murdoch Children's Research Institute, The Royal Children's Hospital Melbourne, Parkville, Melbourne, Australia.
Institute for Molecular Bioscience, The University of Queensland, Brisbane, Australia.

Bryan R Lajoie (BR)

Illumina Inc., San Diego, California.

Joanna Crawford (J)

Institute for Molecular Bioscience, The University of Queensland, Brisbane, Australia.

Asako Takanohashi (A)

Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.

Marzena Walkiewicz (M)

Murdoch Children's Research Institute, The Royal Children's Hospital Melbourne, Parkville, Melbourne, Australia.

Egor Dolzhenko (E)

Illumina Inc., San Diego, California.

Andrew M Gross (AM)

Illumina Inc., San Diego, California.

Vladimir G Gainullin (VG)

Illumina Inc., San Diego, California.

Stephen J Bent (SJ)

Data61, Commonwealth Scientific and Industrial Research Organisation, Brisbane, Australia.

Emma M Jenkinson (EM)

Faculty of Biology, Medicine and Health, School of Biological Sciences, Division of Evolution and Genomic Sciences, University of Manchester, Manchester, United Kingdom.

Sacha Ferdinandusse (S)

Laboratory Genetic Metabolic Diseases, Department of Clinical Chemistry, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam, The Netherlands.

Hans R Waterham (HR)

Laboratory Genetic Metabolic Diseases, Department of Clinical Chemistry, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam, The Netherlands.

Imen Dorboz (I)

INSERM UMR 1141, DHU PROTECT, Université Paris Diderot- Sorbonne, Paris Cité, France.

Enrico Bertini (E)

Unit of Neuromuscular and Neurodegenerative Disorders, Laboratory of Molecular Medicine, Bambino Gesu' Children's Hospital, Rome, Italy.
Genetics and Rare Diseases Research Division, Ospedale Pediatrico Bambino Gesù, 00146, Rome, Italy.

Noriko Miyake (N)

Department of Human Genetics, Yokohama City University Graduate School of Medicine, Fukuura, Kanazawa-ku, Yokohama, 236-0004, Japan.

Nicole I Wolf (NI)

Department of Child Neurology, Emma Children's Hospital, Amsterdam University Medical Centers, Vrije Universiteit Amsterdam and Amsterdam Neuroscience, Amsterdam, The Netherlands.

Truus E M Abbink (TEM)

Department of Child Neurology, Emma Children's Hospital, Amsterdam University Medical Centers, Vrije Universiteit Amsterdam and Amsterdam Neuroscience, Amsterdam, The Netherlands.

Susan M Kirwin (SM)

Molecular Diagnostics Laboratory, Nemours Biomedical Research, Nemours/Alfred I. duPont Hospital for Children, Wilmington, Delaware.

Christina M Tan (CM)

Molecular Diagnostics Laboratory, Nemours Biomedical Research, Nemours/Alfred I. duPont Hospital for Children, Wilmington, Delaware.

Grace M Hobson (GM)

Molecular Diagnostics Laboratory, Nemours Biomedical Research, Nemours/Alfred I. duPont Hospital for Children, Wilmington, Delaware.

Long Guo (L)

Laboratory of Bone and Joint Diseases, RIKEN Center for Integrative Medical Sciences, Tokyo, Japan.

Shiro Ikegawa (S)

Laboratory of Bone and Joint Diseases, RIKEN Center for Integrative Medical Sciences, Tokyo, Japan.

Amy Pizzino (A)

Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.

Johanna L Schmidt (JL)

Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.

Genevieve Bernard (G)

Departments of Neurology and Neurosurgery, Pediatrics, and Human Genetics, McGill University, Montreal, Canada.
Division of Medical Genetics, Montreal Children's Hospital, McGill University Health Center, Montreal, Canada.
Child Health and Human Development Program, Research Institute of the McGill University Health Center, Montreal, Canada.

Raphael Schiffmann (R)

Institute of Metabolic Disease, Baylor Scott & White Research Institute, Dallas, Texas.

Marjo S van der Knaap (MS)

Department of Child Neurology, Emma Children's Hospital, Amsterdam University Medical Centers, Vrije Universiteit Amsterdam and Amsterdam Neuroscience, Amsterdam, The Netherlands.
Department of Functional Genomics, Amsterdam Neuroscience, VU University, Amsterdam, the Netherlands.

Cas Simons (C)

Murdoch Children's Research Institute, The Royal Children's Hospital Melbourne, Parkville, Melbourne, Australia.
Institute for Molecular Bioscience, The University of Queensland, Brisbane, Australia.

Ryan J Taft (RJ)

Illumina Inc., San Diego, California.

Adeline Vanderver (A)

Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.
Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.

Articles similaires

[Redispensing of expensive oral anticancer medicines: a practical application].

Lisanne N van Merendonk, Kübra Akgöl, Bastiaan Nuijen
1.00
Humans Antineoplastic Agents Administration, Oral Drug Costs Counterfeit Drugs

Smoking Cessation and Incident Cardiovascular Disease.

Jun Hwan Cho, Seung Yong Shin, Hoseob Kim et al.
1.00
Humans Male Smoking Cessation Cardiovascular Diseases Female
Humans United States Aged Cross-Sectional Studies Medicare Part C
1.00
Humans Yoga Low Back Pain Female Male

Classifications MeSH