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What, When and How to Measure—Peripheral Biomarkers in Therapy of Huntington’s Disease

Among the main challenges in further advancing therapeutic strategies for Huntington’s disease (HD) is the development of biomarkers which must be applied to assess the efficiency of the treatment. HD is a dreadful neurodegenerative disorder which has its source of pathogenesis in the central nervou...

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Autores principales: Przybyl, Lukasz, Wozna-Wysocka, Magdalena, Kozlowska, Emilia, Fiszer, Agnieszka
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7913877/
https://www.ncbi.nlm.nih.gov/pubmed/33557131
http://dx.doi.org/10.3390/ijms22041561
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author Przybyl, Lukasz
Wozna-Wysocka, Magdalena
Kozlowska, Emilia
Fiszer, Agnieszka
author_facet Przybyl, Lukasz
Wozna-Wysocka, Magdalena
Kozlowska, Emilia
Fiszer, Agnieszka
author_sort Przybyl, Lukasz
collection PubMed
description Among the main challenges in further advancing therapeutic strategies for Huntington’s disease (HD) is the development of biomarkers which must be applied to assess the efficiency of the treatment. HD is a dreadful neurodegenerative disorder which has its source of pathogenesis in the central nervous system (CNS) but is reflected by symptoms in the periphery. Visible symptoms include motor deficits and slight changes in peripheral tissues, which can be used as hallmarks for prognosis of the course of HD, e.g., the onset of the disease symptoms. Knowing how the pathology develops in the context of whole organisms is crucial for the development of therapy which would be the most beneficial for patients, as well as for proposing appropriate biomarkers to monitor disease progression and/or efficiency of treatment. We focus here on molecular peripheral biomarkers which could be used as a measurable outcome of potential therapy. We present and discuss a list of wet biomarkers which have been proposed in recent years to measure pre- and postsymptomatic HD. Interestingly, investigation of peripheral biomarkers in HD can unravel new aspects of the disease pathogenesis. This especially refers to inflammatory proteins or specific immune cells which attract scientific attention in neurodegenerative disorders.
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spelling pubmed-79138772021-02-28 What, When and How to Measure—Peripheral Biomarkers in Therapy of Huntington’s Disease Przybyl, Lukasz Wozna-Wysocka, Magdalena Kozlowska, Emilia Fiszer, Agnieszka Int J Mol Sci Review Among the main challenges in further advancing therapeutic strategies for Huntington’s disease (HD) is the development of biomarkers which must be applied to assess the efficiency of the treatment. HD is a dreadful neurodegenerative disorder which has its source of pathogenesis in the central nervous system (CNS) but is reflected by symptoms in the periphery. Visible symptoms include motor deficits and slight changes in peripheral tissues, which can be used as hallmarks for prognosis of the course of HD, e.g., the onset of the disease symptoms. Knowing how the pathology develops in the context of whole organisms is crucial for the development of therapy which would be the most beneficial for patients, as well as for proposing appropriate biomarkers to monitor disease progression and/or efficiency of treatment. We focus here on molecular peripheral biomarkers which could be used as a measurable outcome of potential therapy. We present and discuss a list of wet biomarkers which have been proposed in recent years to measure pre- and postsymptomatic HD. Interestingly, investigation of peripheral biomarkers in HD can unravel new aspects of the disease pathogenesis. This especially refers to inflammatory proteins or specific immune cells which attract scientific attention in neurodegenerative disorders. MDPI 2021-02-04 /pmc/articles/PMC7913877/ /pubmed/33557131 http://dx.doi.org/10.3390/ijms22041561 Text en © 2021 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (http://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Przybyl, Lukasz
Wozna-Wysocka, Magdalena
Kozlowska, Emilia
Fiszer, Agnieszka
What, When and How to Measure—Peripheral Biomarkers in Therapy of Huntington’s Disease
title What, When and How to Measure—Peripheral Biomarkers in Therapy of Huntington’s Disease
title_full What, When and How to Measure—Peripheral Biomarkers in Therapy of Huntington’s Disease
title_fullStr What, When and How to Measure—Peripheral Biomarkers in Therapy of Huntington’s Disease
title_full_unstemmed What, When and How to Measure—Peripheral Biomarkers in Therapy of Huntington’s Disease
title_short What, When and How to Measure—Peripheral Biomarkers in Therapy of Huntington’s Disease
title_sort what, when and how to measure—peripheral biomarkers in therapy of huntington’s disease
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7913877/
https://www.ncbi.nlm.nih.gov/pubmed/33557131
http://dx.doi.org/10.3390/ijms22041561
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