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Genetically Targeted Clinical Trials in Parkinson’s Disease: Learning from the Successes Made in Oncology

Clinical trials in neurodegenerative disorders have been associated with high rate of failures, while in oncology, the implementation of precision medicine and focus on genetically defined subtypes of disease and targets for drug development have seen an unprecedented success. With more than 20 gene...

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Detalles Bibliográficos
Autores principales: Sjögren, Magnus, Huttunen, Henri J., Svenningsson, Per, Widner, Håkan
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8535305/
https://www.ncbi.nlm.nih.gov/pubmed/34680924
http://dx.doi.org/10.3390/genes12101529
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author Sjögren, Magnus
Huttunen, Henri J.
Svenningsson, Per
Widner, Håkan
author_facet Sjögren, Magnus
Huttunen, Henri J.
Svenningsson, Per
Widner, Håkan
author_sort Sjögren, Magnus
collection PubMed
description Clinical trials in neurodegenerative disorders have been associated with high rate of failures, while in oncology, the implementation of precision medicine and focus on genetically defined subtypes of disease and targets for drug development have seen an unprecedented success. With more than 20 genes associated with Parkinson’s disease (PD), most of which are highly penetrant and often cause early onset or atypical signs and symptoms, and an increasing understanding of the associated pathophysiology culminating in dopaminergic neurodegeneration, applying the technologies and designs into the field of neurodegeneration seems a logical step. This review describes some of the methods used in oncology clinical trials and some attempts in Parkinson’s disease and the potential of further implementing genetics, biomarkers and smart clinical trial designs in this disease area.
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spelling pubmed-85353052021-10-23 Genetically Targeted Clinical Trials in Parkinson’s Disease: Learning from the Successes Made in Oncology Sjögren, Magnus Huttunen, Henri J. Svenningsson, Per Widner, Håkan Genes (Basel) Review Clinical trials in neurodegenerative disorders have been associated with high rate of failures, while in oncology, the implementation of precision medicine and focus on genetically defined subtypes of disease and targets for drug development have seen an unprecedented success. With more than 20 genes associated with Parkinson’s disease (PD), most of which are highly penetrant and often cause early onset or atypical signs and symptoms, and an increasing understanding of the associated pathophysiology culminating in dopaminergic neurodegeneration, applying the technologies and designs into the field of neurodegeneration seems a logical step. This review describes some of the methods used in oncology clinical trials and some attempts in Parkinson’s disease and the potential of further implementing genetics, biomarkers and smart clinical trial designs in this disease area. MDPI 2021-09-28 /pmc/articles/PMC8535305/ /pubmed/34680924 http://dx.doi.org/10.3390/genes12101529 Text en © 2021 by the authors. https://creativecommons.org/licenses/by/4.0/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 (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Sjögren, Magnus
Huttunen, Henri J.
Svenningsson, Per
Widner, Håkan
Genetically Targeted Clinical Trials in Parkinson’s Disease: Learning from the Successes Made in Oncology
title Genetically Targeted Clinical Trials in Parkinson’s Disease: Learning from the Successes Made in Oncology
title_full Genetically Targeted Clinical Trials in Parkinson’s Disease: Learning from the Successes Made in Oncology
title_fullStr Genetically Targeted Clinical Trials in Parkinson’s Disease: Learning from the Successes Made in Oncology
title_full_unstemmed Genetically Targeted Clinical Trials in Parkinson’s Disease: Learning from the Successes Made in Oncology
title_short Genetically Targeted Clinical Trials in Parkinson’s Disease: Learning from the Successes Made in Oncology
title_sort genetically targeted clinical trials in parkinson’s disease: learning from the successes made in oncology
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8535305/
https://www.ncbi.nlm.nih.gov/pubmed/34680924
http://dx.doi.org/10.3390/genes12101529
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