Drug Drop Test: How to Quickly Identify Potential Therapeutic Compounds for Mitochondrial Diseases Using Yeast <i>Saccharomyces cerevisiae</i>

Mitochondrial diseases (MDs) refer to a group of clinically and genetically heterogeneous pathologies characterized by defective mitochondrial function and energy production. Unfortunately, there is no effective treatment for most MDs, and current therapeutic management is limited to relieving sympt...

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Main Authors: Martina Magistrati, Alexandru Ionut Gilea, Maria Carla Gerra, Enrico Baruffini, Cristina Dallabona
Format: Article
Language:English
Published: MDPI AG 2023-06-01
Series:International Journal of Molecular Sciences
Subjects:
Online Access:https://www.mdpi.com/1422-0067/24/13/10696
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author Martina Magistrati
Alexandru Ionut Gilea
Maria Carla Gerra
Enrico Baruffini
Cristina Dallabona
author_facet Martina Magistrati
Alexandru Ionut Gilea
Maria Carla Gerra
Enrico Baruffini
Cristina Dallabona
author_sort Martina Magistrati
collection DOAJ
description Mitochondrial diseases (MDs) refer to a group of clinically and genetically heterogeneous pathologies characterized by defective mitochondrial function and energy production. Unfortunately, there is no effective treatment for most MDs, and current therapeutic management is limited to relieving symptoms. The yeast <i>Saccharomyces cerevisiae</i> has been efficiently used as a model organism to study mitochondria-related disorders thanks to its easy manipulation and well-known mitochondrial biogenesis and metabolism. It has been successfully exploited both to validate alleged pathogenic variants identified in patients and to discover potential beneficial molecules for their treatment. The so-called “drug drop test”, a phenotype-based high-throughput screening, especially if coupled with a drug repurposing approach, allows the identification of molecules with high translational potential in a cost-effective and time-saving manner. In addition to drug identification, <i>S. cerevisiae</i> can be used to point out the drug’s target or pathway. To date, drug drop tests have been successfully carried out for a variety of disease models, leading to very promising results. The most relevant aspect is that studies on more complex model organisms confirmed the effectiveness of the drugs, strengthening the results obtained in yeast and demonstrating the usefulness of this screening as a novel approach to revealing new therapeutic molecules for MDs.
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spelling doaj.art-7a443a589485425d9263e4ff225358392023-11-18T16:42:08ZengMDPI AGInternational Journal of Molecular Sciences1661-65961422-00672023-06-0124131069610.3390/ijms241310696Drug Drop Test: How to Quickly Identify Potential Therapeutic Compounds for Mitochondrial Diseases Using Yeast <i>Saccharomyces cerevisiae</i>Martina Magistrati0Alexandru Ionut Gilea1Maria Carla Gerra2Enrico Baruffini3Cristina Dallabona4Department of Chemistry, Life Sciences and Environmental Sustainability, University of Parma, Parco Area delle Scienze 11/A, 43124 Parma, ItalyDepartment of Chemistry, Life Sciences and Environmental Sustainability, University of Parma, Parco Area delle Scienze 11/A, 43124 Parma, ItalyDepartment of Chemistry, Life Sciences and Environmental Sustainability, University of Parma, Parco Area delle Scienze 11/A, 43124 Parma, ItalyDepartment of Chemistry, Life Sciences and Environmental Sustainability, University of Parma, Parco Area delle Scienze 11/A, 43124 Parma, ItalyDepartment of Chemistry, Life Sciences and Environmental Sustainability, University of Parma, Parco Area delle Scienze 11/A, 43124 Parma, ItalyMitochondrial diseases (MDs) refer to a group of clinically and genetically heterogeneous pathologies characterized by defective mitochondrial function and energy production. Unfortunately, there is no effective treatment for most MDs, and current therapeutic management is limited to relieving symptoms. The yeast <i>Saccharomyces cerevisiae</i> has been efficiently used as a model organism to study mitochondria-related disorders thanks to its easy manipulation and well-known mitochondrial biogenesis and metabolism. It has been successfully exploited both to validate alleged pathogenic variants identified in patients and to discover potential beneficial molecules for their treatment. The so-called “drug drop test”, a phenotype-based high-throughput screening, especially if coupled with a drug repurposing approach, allows the identification of molecules with high translational potential in a cost-effective and time-saving manner. In addition to drug identification, <i>S. cerevisiae</i> can be used to point out the drug’s target or pathway. To date, drug drop tests have been successfully carried out for a variety of disease models, leading to very promising results. The most relevant aspect is that studies on more complex model organisms confirmed the effectiveness of the drugs, strengthening the results obtained in yeast and demonstrating the usefulness of this screening as a novel approach to revealing new therapeutic molecules for MDs.https://www.mdpi.com/1422-0067/24/13/10696drug drop testyeast model<i>Saccharomyces cerevisiae</i>drug repurposingmitochondrial diseases
spellingShingle Martina Magistrati
Alexandru Ionut Gilea
Maria Carla Gerra
Enrico Baruffini
Cristina Dallabona
Drug Drop Test: How to Quickly Identify Potential Therapeutic Compounds for Mitochondrial Diseases Using Yeast <i>Saccharomyces cerevisiae</i>
International Journal of Molecular Sciences
drug drop test
yeast model
<i>Saccharomyces cerevisiae</i>
drug repurposing
mitochondrial diseases
title Drug Drop Test: How to Quickly Identify Potential Therapeutic Compounds for Mitochondrial Diseases Using Yeast <i>Saccharomyces cerevisiae</i>
title_full Drug Drop Test: How to Quickly Identify Potential Therapeutic Compounds for Mitochondrial Diseases Using Yeast <i>Saccharomyces cerevisiae</i>
title_fullStr Drug Drop Test: How to Quickly Identify Potential Therapeutic Compounds for Mitochondrial Diseases Using Yeast <i>Saccharomyces cerevisiae</i>
title_full_unstemmed Drug Drop Test: How to Quickly Identify Potential Therapeutic Compounds for Mitochondrial Diseases Using Yeast <i>Saccharomyces cerevisiae</i>
title_short Drug Drop Test: How to Quickly Identify Potential Therapeutic Compounds for Mitochondrial Diseases Using Yeast <i>Saccharomyces cerevisiae</i>
title_sort drug drop test how to quickly identify potential therapeutic compounds for mitochondrial diseases using yeast i saccharomyces cerevisiae i
topic drug drop test
yeast model
<i>Saccharomyces cerevisiae</i>
drug repurposing
mitochondrial diseases
url https://www.mdpi.com/1422-0067/24/13/10696
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