Zebrafish Models of Prader-Willi Syndrome: Fast Track to Pharmacotherapeutics
Prader-Willi syndrome (PWS) is a rare genetic neurodevelopmental disorder characterized by an insatiable appetite, leading to chronic overeating and obesity. Additional features include short stature, intellectual disability, behavioral problems and incomplete sexual development. Although significan...
| Main Authors: | , , , , , |
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| Format: | Article |
| Language: | English |
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MDPI AG
2016-03-01
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| Series: | Diseases |
| Subjects: | |
| Online Access: | http://www.mdpi.com/2079-9721/4/1/13 |
| _version_ | 1819155428743839744 |
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| author | Emma D. Spikol Caroline E. Laverriere Maya Robnett Gabriela Carter Erin Wolfe Eric Glasgow |
| author_facet | Emma D. Spikol Caroline E. Laverriere Maya Robnett Gabriela Carter Erin Wolfe Eric Glasgow |
| author_sort | Emma D. Spikol |
| collection | DOAJ |
| description | Prader-Willi syndrome (PWS) is a rare genetic neurodevelopmental disorder characterized by an insatiable appetite, leading to chronic overeating and obesity. Additional features include short stature, intellectual disability, behavioral problems and incomplete sexual development. Although significant progress has been made in understanding the genetic basis of PWS, the mechanisms underlying the pathogenesis of the disorder remain poorly understood. Treatment for PWS consists mainly of palliative therapies; curative therapies are sorely needed. Zebrafish, Danio rerio, represent a promising way forward for elucidating physiological problems such as obesity and identifying new pharmacotherapeutic options for PWS. Over the last decade, an increased appreciation for the highly conserved biology among vertebrates and the ability to perform high-throughput drug screening has seen an explosion in the use of zebrafish for disease modeling and drug discovery. Here, we review recent advances in developing zebrafish models of human disease. Aspects of zebrafish genetics and physiology that are relevant to PWS will be discussed, and the advantages and disadvantages of zebrafish models will be contrasted with current animal models for this syndrome. Finally, we will present a paradigm for drug screening in zebrafish that is potentially the fastest route for identifying and delivering curative pharmacotherapies to PWS patients. |
| first_indexed | 2024-12-22T15:36:49Z |
| format | Article |
| id | doaj.art-8f883440ab8547df92f6ed57830f80c8 |
| institution | Directory Open Access Journal |
| issn | 2079-9721 |
| language | English |
| last_indexed | 2024-12-22T15:36:49Z |
| publishDate | 2016-03-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Diseases |
| spelling | doaj.art-8f883440ab8547df92f6ed57830f80c82022-12-21T18:21:14ZengMDPI AGDiseases2079-97212016-03-01411310.3390/diseases4010013diseases4010013Zebrafish Models of Prader-Willi Syndrome: Fast Track to PharmacotherapeuticsEmma D. Spikol0Caroline E. Laverriere1Maya Robnett2Gabriela Carter3Erin Wolfe4Eric Glasgow5Department of Oncology, Georgetown University Medical Center, 4000 Reservoir Road, N.W., Washington, DC 20057, USADepartment of Oncology, Georgetown University Medical Center, 4000 Reservoir Road, N.W., Washington, DC 20057, USADepartment of Oncology, Georgetown University Medical Center, 4000 Reservoir Road, N.W., Washington, DC 20057, USADepartment of Oncology, Georgetown University Medical Center, 4000 Reservoir Road, N.W., Washington, DC 20057, USADepartment of Oncology, Georgetown University Medical Center, 4000 Reservoir Road, N.W., Washington, DC 20057, USADepartment of Oncology, Georgetown University Medical Center, 4000 Reservoir Road, N.W., Washington, DC 20057, USAPrader-Willi syndrome (PWS) is a rare genetic neurodevelopmental disorder characterized by an insatiable appetite, leading to chronic overeating and obesity. Additional features include short stature, intellectual disability, behavioral problems and incomplete sexual development. Although significant progress has been made in understanding the genetic basis of PWS, the mechanisms underlying the pathogenesis of the disorder remain poorly understood. Treatment for PWS consists mainly of palliative therapies; curative therapies are sorely needed. Zebrafish, Danio rerio, represent a promising way forward for elucidating physiological problems such as obesity and identifying new pharmacotherapeutic options for PWS. Over the last decade, an increased appreciation for the highly conserved biology among vertebrates and the ability to perform high-throughput drug screening has seen an explosion in the use of zebrafish for disease modeling and drug discovery. Here, we review recent advances in developing zebrafish models of human disease. Aspects of zebrafish genetics and physiology that are relevant to PWS will be discussed, and the advantages and disadvantages of zebrafish models will be contrasted with current animal models for this syndrome. Finally, we will present a paradigm for drug screening in zebrafish that is potentially the fastest route for identifying and delivering curative pharmacotherapies to PWS patients.http://www.mdpi.com/2079-9721/4/1/13neuroendocrineoxytocinzebrafish |
| spellingShingle | Emma D. Spikol Caroline E. Laverriere Maya Robnett Gabriela Carter Erin Wolfe Eric Glasgow Zebrafish Models of Prader-Willi Syndrome: Fast Track to Pharmacotherapeutics Diseases neuroendocrine oxytocin zebrafish |
| title | Zebrafish Models of Prader-Willi Syndrome: Fast Track to Pharmacotherapeutics |
| title_full | Zebrafish Models of Prader-Willi Syndrome: Fast Track to Pharmacotherapeutics |
| title_fullStr | Zebrafish Models of Prader-Willi Syndrome: Fast Track to Pharmacotherapeutics |
| title_full_unstemmed | Zebrafish Models of Prader-Willi Syndrome: Fast Track to Pharmacotherapeutics |
| title_short | Zebrafish Models of Prader-Willi Syndrome: Fast Track to Pharmacotherapeutics |
| title_sort | zebrafish models of prader willi syndrome fast track to pharmacotherapeutics |
| topic | neuroendocrine oxytocin zebrafish |
| url | http://www.mdpi.com/2079-9721/4/1/13 |
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