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...
Main Authors: | , , , , |
---|---|
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 |
_version_ | 1797591558540754944 |
---|---|
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. |
first_indexed | 2024-03-11T01:40:07Z |
format | Article |
id | doaj.art-7a443a589485425d9263e4ff22535839 |
institution | Directory Open Access Journal |
issn | 1661-6596 1422-0067 |
language | English |
last_indexed | 2024-03-11T01:40:07Z |
publishDate | 2023-06-01 |
publisher | MDPI AG |
record_format | Article |
series | International Journal of Molecular Sciences |
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 |
work_keys_str_mv | AT martinamagistrati drugdroptesthowtoquicklyidentifypotentialtherapeuticcompoundsformitochondrialdiseasesusingyeastisaccharomycescerevisiaei AT alexandruionutgilea drugdroptesthowtoquicklyidentifypotentialtherapeuticcompoundsformitochondrialdiseasesusingyeastisaccharomycescerevisiaei AT mariacarlagerra drugdroptesthowtoquicklyidentifypotentialtherapeuticcompoundsformitochondrialdiseasesusingyeastisaccharomycescerevisiaei AT enricobaruffini drugdroptesthowtoquicklyidentifypotentialtherapeuticcompoundsformitochondrialdiseasesusingyeastisaccharomycescerevisiaei AT cristinadallabona drugdroptesthowtoquicklyidentifypotentialtherapeuticcompoundsformitochondrialdiseasesusingyeastisaccharomycescerevisiaei |