Zebrafish Models of Rare Neurological Diseases like Spinocerebellar Ataxias (SCAs): Advantages and Limitations
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 therapi...
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| Format: | Article |
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MDPI AG
2023-10-01
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| Series: | Biology |
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| Online Access: | https://www.mdpi.com/2079-7737/12/10/1322 |
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| author | Sreeja Sarasamma Anwarul Karim James P. Orengo |
| author_facet | Sreeja Sarasamma Anwarul Karim James P. Orengo |
| author_sort | Sreeja Sarasamma |
| collection | DOAJ |
| description | 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 (<i>Danio rerio</i>) 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. |
| first_indexed | 2024-03-10T21:25:31Z |
| format | Article |
| id | doaj.art-7a22893a72b14fad9af12a8f7a973286 |
| institution | Directory Open Access Journal |
| issn | 2079-7737 |
| language | English |
| last_indexed | 2024-03-10T21:25:31Z |
| publishDate | 2023-10-01 |
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| spelling | doaj.art-7a22893a72b14fad9af12a8f7a9732862023-11-19T15:43:40ZengMDPI AGBiology2079-77372023-10-011210132210.3390/biology12101322Zebrafish Models of Rare Neurological Diseases like Spinocerebellar Ataxias (SCAs): Advantages and LimitationsSreeja Sarasamma0Anwarul Karim1James P. Orengo2Departments of Neurology and Neuroscience, Baylor College of Medicine, Houston, TX 77030, USASchool of Clinical Medicine, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Hong Kong SAR, ChinaDepartments of Neurology and Neuroscience, Baylor College of Medicine, Houston, TX 77030, USASpinocerebellar 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 (<i>Danio rerio</i>) 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.https://www.mdpi.com/2079-7737/12/10/1322zebrafishneurodegenerationspinocerebellar ataxiabehavioral assaysdrug discovery |
| spellingShingle | Sreeja Sarasamma Anwarul Karim James P. Orengo Zebrafish Models of Rare Neurological Diseases like Spinocerebellar Ataxias (SCAs): Advantages and Limitations Biology zebrafish neurodegeneration spinocerebellar ataxia behavioral assays drug discovery |
| 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 | zebrafish neurodegeneration spinocerebellar ataxia behavioral assays drug discovery |
| url | https://www.mdpi.com/2079-7737/12/10/1322 |
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