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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...
Autores principales: | , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2021
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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. |
format | Online Article Text |
id | pubmed-8535305 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
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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