Implementation of Exome Sequencing in Clinical Practice for Neurological Disorders.

Parkinson ataxia autism spectrum disorder dystonia epilepsy neurodevelopmental disorders neurological disorders spastic paraplegia whole exome sequencing

Journal

Genes
ISSN: 2073-4425
Titre abrégé: Genes (Basel)
Pays: Switzerland
ID NLM: 101551097

Informations de publication

Date de publication:
28 03 2023
Historique:
received: 20 02 2023
revised: 21 03 2023
accepted: 25 03 2023
medline: 1 5 2023
pubmed: 28 4 2023
entrez: 28 4 2023
Statut: epublish

Résumé

Neurological disorders (ND) are diseases that affect the brain and the central and autonomic nervous systems, such as neurodevelopmental disorders, cerebellar ataxias, Parkinson's disease, or epilepsies. Nowadays, recommendations of the American College of Medical Genetics and Genomics strongly recommend applying next generation sequencing (NGS) as a first-line test in patients with these disorders. Whole exome sequencing (WES) is widely regarded as the current technology of choice for diagnosing monogenic ND. The introduction of NGS allows for rapid and inexpensive large-scale genomic analysis and has led to enormous progress in deciphering monogenic forms of various genetic diseases. The simultaneous analysis of several potentially mutated genes improves the diagnostic process, making it faster and more efficient. The main aim of this report is to discuss the impact and advantages of the implementation of WES into the clinical diagnosis and management of ND. Therefore, we have performed a retrospective evaluation of WES application in 209 cases referred to the Department of Biochemistry and Molecular Genetics of the Hospital Clinic of Barcelona for WES sequencing derived from neurologists or clinical geneticists. In addition, we have further discussed some important facts regarding classification criteria for pathogenicity of rare variants, variants of unknown significance, deleterious variants, different clinical phenotypes, or frequency of actionable secondary findings. Different studies have shown that WES implementation establish diagnostic rate around 32% in ND and the continuous molecular diagnosis is essential to solve the remaining cases.

Identifiants

pubmed: 37107571
pii: genes14040813
doi: 10.3390/genes14040813
pmc: PMC10137364
pii:
doi:

Types de publication

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

Langues

eng

Sous-ensembles de citation

IM

Références

Mol Med. 2022 Mar 26;28(1):38
pubmed: 35346031
Eur J Hum Genet. 2021 Mar;29(3):365-377
pubmed: 33223530
Genet Med. 2013 Jul;15(7):565-74
pubmed: 23788249
Parkinsonism Relat Disord. 2019 Dec;69:111-118
pubmed: 31731261
J Neurol Sci. 2021 Jan 15;420:117260
pubmed: 33310205
F1000Res. 2020 Jan 29;9:63
pubmed: 32269765
Hum Mutat. 2016 Jun;37(6):564-9
pubmed: 26931183
Nucleic Acids Res. 2016 Jan 4;44(D1):D862-8
pubmed: 26582918
JAMA. 2014 Nov 12;312(18):1880-7
pubmed: 25326637
Orphanet J Rare Dis. 2022 Feb 19;17(1):60
pubmed: 35183220
J Clin Invest. 2021 Jan 4;131(1):
pubmed: 33001864
J Mol Diagn. 2022 May;24(5):529-542
pubmed: 35569879
Hum Mutat. 2015 Oct;36(10):928-30
pubmed: 26220891
Genome Biol. 2020 Jun 17;21(1):145
pubmed: 32552793
Hum Mutat. 2020 Feb;41(2):487-501
pubmed: 31692161
Genet Med. 2021 Aug;23(8):1381-1390
pubmed: 34012068
Genome Med. 2022 Apr 5;14(1):38
pubmed: 35379322
Am J Hum Genet. 2010 May 14;86(5):749-64
pubmed: 20466091
Genet Med. 2016 Jul;18(7):696-704
pubmed: 26633542
Life (Basel). 2021 Apr 19;11(4):
pubmed: 33921670
Epilepsia. 2020 Feb;61(2):249-258
pubmed: 31957018
Nat Rev Neurol. 2013 Aug;9(8):445-54
pubmed: 23857047
Genet Med. 2020 Mar;22(3):490-499
pubmed: 31607746
Am J Med Genet A. 2003 Nov 15;123A(1):29-32
pubmed: 14556243
Genes (Basel). 2021 Apr 12;12(4):
pubmed: 33921338
Lancet Neurol. 2020 Nov;19(11):908-918
pubmed: 33098801
Eur J Hum Genet. 2021 Jan;29(1):173-183
pubmed: 32862204
Genet Med. 2015 May;17(5):405-24
pubmed: 25741868
Genet Med. 2021 Aug;23(8):1391-1398
pubmed: 34012069
Genet Med. 2021 Nov;23(11):2029-2037
pubmed: 34211152
NPJ Genom Med. 2020 Oct 23;5:47
pubmed: 33110627
Hum Mutat. 2003 Jun;21(6):577-81
pubmed: 12754702
Eur J Med Genet. 2017 May;60(5):265-267
pubmed: 28286254
Mol Genet Metab Rep. 2019 Mar 01;19:100464
pubmed: 30891420
Lancet Neurol. 2019 May;18(5):492-503
pubmed: 30981321
Eur J Hum Genet. 2016 Jan;24(1):2-5
pubmed: 26508566
Genet Med. 2017 Feb;19(2):249-255
pubmed: 27854360

Auteurs

María Isabel Alvarez-Mora (MI)

Biochemistry and Molecular Genetics Department, Hospital Clinic of Barcelona, IDIBAPS (Institut de Investigacions Biomèdiques August Pi I Sunyer), 08036 Barcelona, Spain.
CIBER of Rare Diseases (CIBERER), 08036 Barcelona, Spain.

Laia Rodríguez-Revenga (L)

Biochemistry and Molecular Genetics Department, Hospital Clinic of Barcelona, IDIBAPS (Institut de Investigacions Biomèdiques August Pi I Sunyer), 08036 Barcelona, Spain.
CIBER of Rare Diseases (CIBERER), 08036 Barcelona, Spain.

Meritxell Jodar (M)

Biochemistry and Molecular Genetics Department, Hospital Clinic of Barcelona, IDIBAPS (Institut de Investigacions Biomèdiques August Pi I Sunyer), 08036 Barcelona, Spain.
Molecular Biology of Reproduction and Development Research Group, Department of Biomedical Sciences, Faculty of Medicine, Universitat de Barcelona, 08036 Barcelona, Spain.

Miriam Potrony (M)

Biochemistry and Molecular Genetics Department, Hospital Clinic of Barcelona, IDIBAPS (Institut de Investigacions Biomèdiques August Pi I Sunyer), 08036 Barcelona, Spain.
CIBER of Rare Diseases (CIBERER), 08036 Barcelona, Spain.

Aurora Sanchez (A)

Biochemistry and Molecular Genetics Department, Hospital Clinic of Barcelona, IDIBAPS (Institut de Investigacions Biomèdiques August Pi I Sunyer), 08036 Barcelona, Spain.
CIBER of Rare Diseases (CIBERER), 08036 Barcelona, Spain.

Celia Badenas (C)

Biochemistry and Molecular Genetics Department, Hospital Clinic of Barcelona, IDIBAPS (Institut de Investigacions Biomèdiques August Pi I Sunyer), 08036 Barcelona, Spain.
CIBER of Rare Diseases (CIBERER), 08036 Barcelona, Spain.

Josep Oriola (J)

Biochemistry and Molecular Genetics Department, Hospital Clinic of Barcelona, IDIBAPS (Institut de Investigacions Biomèdiques August Pi I Sunyer), 08036 Barcelona, Spain.

José Luis Villanueva-Cañas (JL)

Molecular Biology CORE (CDB), Hospital Clínic de Barcelona, 08036 Barcelona, Spain.

Esteban Muñoz (E)

Parkinson's Disease & Movement Disorders Unit, Neurology Service, Hospital Clínic Universitari de Barcelona, IDIBAPS, CIBERNED (CB06/05/0018-ISCIII), ERN-RND, Institut Clínic de Neurociències UBNeuro (Maria de Maeztu Excellence Centre), Universitat de Barcelona, 08036 Barcelona, Spain.

Francesc Valldeoriola (F)

Parkinson's Disease & Movement Disorders Unit, Neurology Service, Hospital Clínic Universitari de Barcelona, IDIBAPS, CIBERNED (CB06/05/0018-ISCIII), ERN-RND, Institut Clínic de Neurociències UBNeuro (Maria de Maeztu Excellence Centre), Universitat de Barcelona, 08036 Barcelona, Spain.

Ana Cámara (A)

Parkinson's Disease & Movement Disorders Unit, Neurology Service, Hospital Clínic Universitari de Barcelona, IDIBAPS, CIBERNED (CB06/05/0018-ISCIII), ERN-RND, Institut Clínic de Neurociències UBNeuro (Maria de Maeztu Excellence Centre), Universitat de Barcelona, 08036 Barcelona, Spain.

Yaroslau Compta (Y)

Parkinson's Disease & Movement Disorders Unit, Neurology Service, Hospital Clínic Universitari de Barcelona, IDIBAPS, CIBERNED (CB06/05/0018-ISCIII), ERN-RND, Institut Clínic de Neurociències UBNeuro (Maria de Maeztu Excellence Centre), Universitat de Barcelona, 08036 Barcelona, Spain.

Mar Carreño (M)

Epilepsy Unit, Department of Neurology, Hospital Clinic, 08036 Barcelona, Spain.

María Jose Martí (MJ)

Parkinson's Disease & Movement Disorders Unit, Neurology Service, Hospital Clínic Universitari de Barcelona, IDIBAPS, CIBERNED (CB06/05/0018-ISCIII), ERN-RND, Institut Clínic de Neurociències UBNeuro (Maria de Maeztu Excellence Centre), Universitat de Barcelona, 08036 Barcelona, Spain.

Raquel Sánchez-Valle (R)

Neurology Department, Clinical Institute of Neurosciences, Hospital Clinic of Barcelona, 08036 Barcelona, Spain.
Biomedical Research Institute August Pi i Sunyer (IDIBAPS), Hospital Clinic of Barcelona, 08036 Barcelona, Spain.

Irene Madrigal (I)

Biochemistry and Molecular Genetics Department, Hospital Clinic of Barcelona, IDIBAPS (Institut de Investigacions Biomèdiques August Pi I Sunyer), 08036 Barcelona, Spain.
CIBER of Rare Diseases (CIBERER), 08036 Barcelona, Spain.

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