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Epilepsy Genetics and Precision Medicine in Adults: A New Landscape for Developmental and Epileptic Encephalopathies

This review aims to provide an updated perspective of epilepsy genetics and precision medicine in adult patients, with special focus on developmental and epileptic encephalopathies (DEEs), covering relevant and controversial issues, such as defining candidates for genetic testing, which genetic test...

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Autores principales: Beltrán-Corbellini, Álvaro, Aledo-Serrano, Ángel, Møller, Rikke S., Pérez-Palma, Eduardo, García-Morales, Irene, Toledano, Rafael, Gil-Nagel, Antonio
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8891166/
https://www.ncbi.nlm.nih.gov/pubmed/35250806
http://dx.doi.org/10.3389/fneur.2022.777115
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author Beltrán-Corbellini, Álvaro
Aledo-Serrano, Ángel
Møller, Rikke S.
Pérez-Palma, Eduardo
García-Morales, Irene
Toledano, Rafael
Gil-Nagel, Antonio
author_facet Beltrán-Corbellini, Álvaro
Aledo-Serrano, Ángel
Møller, Rikke S.
Pérez-Palma, Eduardo
García-Morales, Irene
Toledano, Rafael
Gil-Nagel, Antonio
author_sort Beltrán-Corbellini, Álvaro
collection PubMed
description This review aims to provide an updated perspective of epilepsy genetics and precision medicine in adult patients, with special focus on developmental and epileptic encephalopathies (DEEs), covering relevant and controversial issues, such as defining candidates for genetic testing, which genetic tests to request and how to interpret them. A literature review was conducted, including findings in the discussion and recommendations. DEEs are wide and phenotypically heterogeneous electroclinical syndromes. They generally have a pediatric presentation, but patients frequently reach adulthood still undiagnosed. Identifying the etiology is essential, because there lies the key for precision medicine. Phenotypes modify according to age, and although deep phenotyping has allowed to outline certain entities, genotype-phenotype correlations are still poor, commonly leading to long-lasting diagnostic odysseys and ineffective therapies. Recent adult series show that the target patients to be identified for genetic testing are those with epilepsy and different risk factors. The clinician should take active part in the assessment of the pathogenicity of the variants detected, especially concerning variants of uncertain significance. An accurate diagnosis implies precision medicine, meaning genetic counseling, prognosis, possible future therapies, and a reduction of iatrogeny. Up to date, there are a few tens of gene mutations with additional concrete treatments, including those with restrictive/substitutive therapies, those with therapies modifying signaling pathways, and channelopathies, that are worth to be assessed in adults. Further research is needed regarding phenotyping of adult syndromes, early diagnosis, and the development of targeted therapies.
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spelling pubmed-88911662022-03-04 Epilepsy Genetics and Precision Medicine in Adults: A New Landscape for Developmental and Epileptic Encephalopathies Beltrán-Corbellini, Álvaro Aledo-Serrano, Ángel Møller, Rikke S. Pérez-Palma, Eduardo García-Morales, Irene Toledano, Rafael Gil-Nagel, Antonio Front Neurol Neurology This review aims to provide an updated perspective of epilepsy genetics and precision medicine in adult patients, with special focus on developmental and epileptic encephalopathies (DEEs), covering relevant and controversial issues, such as defining candidates for genetic testing, which genetic tests to request and how to interpret them. A literature review was conducted, including findings in the discussion and recommendations. DEEs are wide and phenotypically heterogeneous electroclinical syndromes. They generally have a pediatric presentation, but patients frequently reach adulthood still undiagnosed. Identifying the etiology is essential, because there lies the key for precision medicine. Phenotypes modify according to age, and although deep phenotyping has allowed to outline certain entities, genotype-phenotype correlations are still poor, commonly leading to long-lasting diagnostic odysseys and ineffective therapies. Recent adult series show that the target patients to be identified for genetic testing are those with epilepsy and different risk factors. The clinician should take active part in the assessment of the pathogenicity of the variants detected, especially concerning variants of uncertain significance. An accurate diagnosis implies precision medicine, meaning genetic counseling, prognosis, possible future therapies, and a reduction of iatrogeny. Up to date, there are a few tens of gene mutations with additional concrete treatments, including those with restrictive/substitutive therapies, those with therapies modifying signaling pathways, and channelopathies, that are worth to be assessed in adults. Further research is needed regarding phenotyping of adult syndromes, early diagnosis, and the development of targeted therapies. Frontiers Media S.A. 2022-02-17 /pmc/articles/PMC8891166/ /pubmed/35250806 http://dx.doi.org/10.3389/fneur.2022.777115 Text en Copyright © 2022 Beltrán-Corbellini, Aledo-Serrano, Møller, Pérez-Palma, García-Morales, Toledano and Gil-Nagel. https://creativecommons.org/licenses/by/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
spellingShingle Neurology
Beltrán-Corbellini, Álvaro
Aledo-Serrano, Ángel
Møller, Rikke S.
Pérez-Palma, Eduardo
García-Morales, Irene
Toledano, Rafael
Gil-Nagel, Antonio
Epilepsy Genetics and Precision Medicine in Adults: A New Landscape for Developmental and Epileptic Encephalopathies
title Epilepsy Genetics and Precision Medicine in Adults: A New Landscape for Developmental and Epileptic Encephalopathies
title_full Epilepsy Genetics and Precision Medicine in Adults: A New Landscape for Developmental and Epileptic Encephalopathies
title_fullStr Epilepsy Genetics and Precision Medicine in Adults: A New Landscape for Developmental and Epileptic Encephalopathies
title_full_unstemmed Epilepsy Genetics and Precision Medicine in Adults: A New Landscape for Developmental and Epileptic Encephalopathies
title_short Epilepsy Genetics and Precision Medicine in Adults: A New Landscape for Developmental and Epileptic Encephalopathies
title_sort epilepsy genetics and precision medicine in adults: a new landscape for developmental and epileptic encephalopathies
topic Neurology
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8891166/
https://www.ncbi.nlm.nih.gov/pubmed/35250806
http://dx.doi.org/10.3389/fneur.2022.777115
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