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The Promise and Challenges of Developing miRNA-Based Therapeutics for Parkinson’s Disease
MicroRNAs (miRNAs) are small double-stranded RNAs that exert a fine-tuning sequence-specific regulation of cell transcriptome. While one unique miRNA regulates hundreds of mRNAs, each mRNA molecule is commonly regulated by various miRNAs that bind to complementary sequences at 3’-untranslated region...
Autores principales: | , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7226753/ https://www.ncbi.nlm.nih.gov/pubmed/32244357 http://dx.doi.org/10.3390/cells9040841 |
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author | Titze-de-Almeida, Simoneide S. Soto-Sánchez, Cristina Fernandez, Eduardo Koprich, James B. Brotchie, Jonathan M. Titze-de-Almeida, Ricardo |
author_facet | Titze-de-Almeida, Simoneide S. Soto-Sánchez, Cristina Fernandez, Eduardo Koprich, James B. Brotchie, Jonathan M. Titze-de-Almeida, Ricardo |
author_sort | Titze-de-Almeida, Simoneide S. |
collection | PubMed |
description | MicroRNAs (miRNAs) are small double-stranded RNAs that exert a fine-tuning sequence-specific regulation of cell transcriptome. While one unique miRNA regulates hundreds of mRNAs, each mRNA molecule is commonly regulated by various miRNAs that bind to complementary sequences at 3’-untranslated regions for triggering the mechanism of RNA interference. Unfortunately, dysregulated miRNAs play critical roles in many disorders, including Parkinson’s disease (PD), the second most prevalent neurodegenerative disease in the world. Treatment of this slowly, progressive, and yet incurable pathology challenges neurologists. In addition to L-DOPA that restores dopaminergic transmission and ameliorate motor signs (i.e., bradykinesia, rigidity, tremors), patients commonly receive medication for mood disorders and autonomic dysfunctions. However, the effectiveness of L-DOPA declines over time, and the L-DOPA-induced dyskinesias commonly appear and become highly disabling. The discovery of more effective therapies capable of slowing disease progression –a neuroprotective agent–remains a critical need in PD. The present review focus on miRNAs as promising drug targets for PD, examining their role in underlying mechanisms of the disease, the strategies for controlling aberrant expressions, and, finally, the current technologies for translating these small molecules from bench to clinics. |
format | Online Article Text |
id | pubmed-7226753 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2020 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
spelling | pubmed-72267532020-05-18 The Promise and Challenges of Developing miRNA-Based Therapeutics for Parkinson’s Disease Titze-de-Almeida, Simoneide S. Soto-Sánchez, Cristina Fernandez, Eduardo Koprich, James B. Brotchie, Jonathan M. Titze-de-Almeida, Ricardo Cells Review MicroRNAs (miRNAs) are small double-stranded RNAs that exert a fine-tuning sequence-specific regulation of cell transcriptome. While one unique miRNA regulates hundreds of mRNAs, each mRNA molecule is commonly regulated by various miRNAs that bind to complementary sequences at 3’-untranslated regions for triggering the mechanism of RNA interference. Unfortunately, dysregulated miRNAs play critical roles in many disorders, including Parkinson’s disease (PD), the second most prevalent neurodegenerative disease in the world. Treatment of this slowly, progressive, and yet incurable pathology challenges neurologists. In addition to L-DOPA that restores dopaminergic transmission and ameliorate motor signs (i.e., bradykinesia, rigidity, tremors), patients commonly receive medication for mood disorders and autonomic dysfunctions. However, the effectiveness of L-DOPA declines over time, and the L-DOPA-induced dyskinesias commonly appear and become highly disabling. The discovery of more effective therapies capable of slowing disease progression –a neuroprotective agent–remains a critical need in PD. The present review focus on miRNAs as promising drug targets for PD, examining their role in underlying mechanisms of the disease, the strategies for controlling aberrant expressions, and, finally, the current technologies for translating these small molecules from bench to clinics. MDPI 2020-03-31 /pmc/articles/PMC7226753/ /pubmed/32244357 http://dx.doi.org/10.3390/cells9040841 Text en © 2020 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 Titze-de-Almeida, Simoneide S. Soto-Sánchez, Cristina Fernandez, Eduardo Koprich, James B. Brotchie, Jonathan M. Titze-de-Almeida, Ricardo The Promise and Challenges of Developing miRNA-Based Therapeutics for Parkinson’s Disease |
title | The Promise and Challenges of Developing miRNA-Based Therapeutics for Parkinson’s Disease |
title_full | The Promise and Challenges of Developing miRNA-Based Therapeutics for Parkinson’s Disease |
title_fullStr | The Promise and Challenges of Developing miRNA-Based Therapeutics for Parkinson’s Disease |
title_full_unstemmed | The Promise and Challenges of Developing miRNA-Based Therapeutics for Parkinson’s Disease |
title_short | The Promise and Challenges of Developing miRNA-Based Therapeutics for Parkinson’s Disease |
title_sort | promise and challenges of developing mirna-based therapeutics for parkinson’s disease |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7226753/ https://www.ncbi.nlm.nih.gov/pubmed/32244357 http://dx.doi.org/10.3390/cells9040841 |
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