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From Multi-Omics Approaches to Precision Medicine in Amyotrophic Lateral Sclerosis

Amyotrophic lateral sclerosis (ALS) is a devastating and fatal neurodegenerative disorder, caused by the degeneration of upper and lower motor neurons for which there is no truly effective cure. The lack of successful treatments can be well explained by the complex and heterogeneous nature of ALS, w...

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Autores principales: Morello, Giovanna, Salomone, Salvatore, D’Agata, Velia, Conforti, Francesca Luisa, Cavallaro, Sebastiano
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7661549/
https://www.ncbi.nlm.nih.gov/pubmed/33192262
http://dx.doi.org/10.3389/fnins.2020.577755
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author Morello, Giovanna
Salomone, Salvatore
D’Agata, Velia
Conforti, Francesca Luisa
Cavallaro, Sebastiano
author_facet Morello, Giovanna
Salomone, Salvatore
D’Agata, Velia
Conforti, Francesca Luisa
Cavallaro, Sebastiano
author_sort Morello, Giovanna
collection PubMed
description Amyotrophic lateral sclerosis (ALS) is a devastating and fatal neurodegenerative disorder, caused by the degeneration of upper and lower motor neurons for which there is no truly effective cure. The lack of successful treatments can be well explained by the complex and heterogeneous nature of ALS, with patients displaying widely distinct clinical features and progression patterns, and distinct molecular mechanisms underlying the phenotypic heterogeneity. Thus, stratifying ALS patients into consistent and clinically relevant subgroups can be of great value for the development of new precision diagnostics and targeted therapeutics for ALS patients. In the last years, the use and integration of high-throughput “omics” approaches have dramatically changed our thinking about ALS, improving our understanding of the complex molecular architecture of ALS, distinguishing distinct patient subtypes and providing a rational foundation for the discovery of biomarkers and new individualized treatments. In this review, we discuss the most significant contributions of omics technologies in unraveling the biological heterogeneity of ALS, highlighting how these approaches are revealing diagnostic, prognostic and therapeutic targets for future personalized interventions.
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spelling pubmed-76615492020-11-13 From Multi-Omics Approaches to Precision Medicine in Amyotrophic Lateral Sclerosis Morello, Giovanna Salomone, Salvatore D’Agata, Velia Conforti, Francesca Luisa Cavallaro, Sebastiano Front Neurosci Neuroscience Amyotrophic lateral sclerosis (ALS) is a devastating and fatal neurodegenerative disorder, caused by the degeneration of upper and lower motor neurons for which there is no truly effective cure. The lack of successful treatments can be well explained by the complex and heterogeneous nature of ALS, with patients displaying widely distinct clinical features and progression patterns, and distinct molecular mechanisms underlying the phenotypic heterogeneity. Thus, stratifying ALS patients into consistent and clinically relevant subgroups can be of great value for the development of new precision diagnostics and targeted therapeutics for ALS patients. In the last years, the use and integration of high-throughput “omics” approaches have dramatically changed our thinking about ALS, improving our understanding of the complex molecular architecture of ALS, distinguishing distinct patient subtypes and providing a rational foundation for the discovery of biomarkers and new individualized treatments. In this review, we discuss the most significant contributions of omics technologies in unraveling the biological heterogeneity of ALS, highlighting how these approaches are revealing diagnostic, prognostic and therapeutic targets for future personalized interventions. Frontiers Media S.A. 2020-10-30 /pmc/articles/PMC7661549/ /pubmed/33192262 http://dx.doi.org/10.3389/fnins.2020.577755 Text en Copyright © 2020 Morello, Salomone, D’Agata, Conforti and Cavallaro. http://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 Neuroscience
Morello, Giovanna
Salomone, Salvatore
D’Agata, Velia
Conforti, Francesca Luisa
Cavallaro, Sebastiano
From Multi-Omics Approaches to Precision Medicine in Amyotrophic Lateral Sclerosis
title From Multi-Omics Approaches to Precision Medicine in Amyotrophic Lateral Sclerosis
title_full From Multi-Omics Approaches to Precision Medicine in Amyotrophic Lateral Sclerosis
title_fullStr From Multi-Omics Approaches to Precision Medicine in Amyotrophic Lateral Sclerosis
title_full_unstemmed From Multi-Omics Approaches to Precision Medicine in Amyotrophic Lateral Sclerosis
title_short From Multi-Omics Approaches to Precision Medicine in Amyotrophic Lateral Sclerosis
title_sort from multi-omics approaches to precision medicine in amyotrophic lateral sclerosis
topic Neuroscience
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7661549/
https://www.ncbi.nlm.nih.gov/pubmed/33192262
http://dx.doi.org/10.3389/fnins.2020.577755
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