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Zebrafish Models of Rare Neurological Diseases like Spinocerebellar Ataxias (SCAs): Advantages and Limitations

SIMPLE SUMMARY: Zebrafish can be used as an effective model for studying various genetic neurological disorders, such as spinocerebellar ataxias. They are cost effective, easy to genetically manipulate, transparent in the larvae stage allowing for in vivo imaging, and amenable to rapid compound scre...

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Autores principales: Sarasamma, Sreeja, Karim, Anwarul, Orengo, James P.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10604122/
https://www.ncbi.nlm.nih.gov/pubmed/37887032
http://dx.doi.org/10.3390/biology12101322
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author Sarasamma, Sreeja
Karim, Anwarul
Orengo, James P.
author_facet Sarasamma, Sreeja
Karim, Anwarul
Orengo, James P.
author_sort Sarasamma, Sreeja
collection PubMed
description SIMPLE SUMMARY: Zebrafish can be used as an effective model for studying various genetic neurological disorders, such as spinocerebellar ataxias. They are cost effective, easy to genetically manipulate, transparent in the larvae stage allowing for in vivo imaging, and amenable to rapid compound screens. Zebrafish can link the gap between neuro-specific drug discovery and clinical trials. ABSTRACT: Spinocerebellar ataxia (SCA) is a heterogeneous group of rare familial neurodegenerative disorders that share the key feature of cerebellar ataxia. Clinical heterogeneity, diverse gene mutations and complex neuropathology pose significant challenges for developing effective disease-modifying therapies in SCAs. Without a deep understanding of the molecular mechanisms involved for each SCA, we cannot succeed in developing targeted therapies. Animal models are our best tool to address these issues and several have been generated to study the pathological conditions of SCAs. Among them, zebrafish (Danio rerio) models are emerging as a powerful tool for in vivo study of SCAs, as well as rapid drug screens. In this review, we will summarize recent progress in using zebrafish to study the pathology of SCAs. We will discuss recent advancements on how zebrafish models can further clarify underlying genetic, neuroanatomical, and behavioral pathogenic mechanisms of disease. We highlight their usefulness in rapid drug discovery and large screens. Finally, we will discuss the advantages and limitations of this in vivo model to develop tailored therapeutic strategies for SCA.
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spelling pubmed-106041222023-10-28 Zebrafish Models of Rare Neurological Diseases like Spinocerebellar Ataxias (SCAs): Advantages and Limitations Sarasamma, Sreeja Karim, Anwarul Orengo, James P. Biology (Basel) Review SIMPLE SUMMARY: Zebrafish can be used as an effective model for studying various genetic neurological disorders, such as spinocerebellar ataxias. They are cost effective, easy to genetically manipulate, transparent in the larvae stage allowing for in vivo imaging, and amenable to rapid compound screens. Zebrafish can link the gap between neuro-specific drug discovery and clinical trials. ABSTRACT: Spinocerebellar ataxia (SCA) is a heterogeneous group of rare familial neurodegenerative disorders that share the key feature of cerebellar ataxia. Clinical heterogeneity, diverse gene mutations and complex neuropathology pose significant challenges for developing effective disease-modifying therapies in SCAs. Without a deep understanding of the molecular mechanisms involved for each SCA, we cannot succeed in developing targeted therapies. Animal models are our best tool to address these issues and several have been generated to study the pathological conditions of SCAs. Among them, zebrafish (Danio rerio) models are emerging as a powerful tool for in vivo study of SCAs, as well as rapid drug screens. In this review, we will summarize recent progress in using zebrafish to study the pathology of SCAs. We will discuss recent advancements on how zebrafish models can further clarify underlying genetic, neuroanatomical, and behavioral pathogenic mechanisms of disease. We highlight their usefulness in rapid drug discovery and large screens. Finally, we will discuss the advantages and limitations of this in vivo model to develop tailored therapeutic strategies for SCA. MDPI 2023-10-10 /pmc/articles/PMC10604122/ /pubmed/37887032 http://dx.doi.org/10.3390/biology12101322 Text en © 2023 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
Sarasamma, Sreeja
Karim, Anwarul
Orengo, James P.
Zebrafish Models of Rare Neurological Diseases like Spinocerebellar Ataxias (SCAs): Advantages and Limitations
title Zebrafish Models of Rare Neurological Diseases like Spinocerebellar Ataxias (SCAs): Advantages and Limitations
title_full Zebrafish Models of Rare Neurological Diseases like Spinocerebellar Ataxias (SCAs): Advantages and Limitations
title_fullStr Zebrafish Models of Rare Neurological Diseases like Spinocerebellar Ataxias (SCAs): Advantages and Limitations
title_full_unstemmed Zebrafish Models of Rare Neurological Diseases like Spinocerebellar Ataxias (SCAs): Advantages and Limitations
title_short Zebrafish Models of Rare Neurological Diseases like Spinocerebellar Ataxias (SCAs): Advantages and Limitations
title_sort zebrafish models of rare neurological diseases like spinocerebellar ataxias (scas): advantages and limitations
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10604122/
https://www.ncbi.nlm.nih.gov/pubmed/37887032
http://dx.doi.org/10.3390/biology12101322
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