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Zebrafish Models of Rare Hereditary Pediatric Diseases

Department of Genetics, ELTE Eötvös Loránd University, 1117 Budapest, Hungary
MTA-SE Lendület Nephrogenetic Laboratory, 1083 Budapest, Hungary
Ist Department of Pediatrics, Semmelweis University, 1083 Budapest, Hungary
Authors to whom correspondence should be addressed.
Diseases 2018, 6(2), 43;
Received: 27 April 2018 / Revised: 17 May 2018 / Accepted: 19 May 2018 / Published: 22 May 2018
(This article belongs to the Special Issue Pediatric Diseases)
PDF [1178 KB, uploaded 22 May 2018]


Recent advances in sequencing technologies have made it significantly easier to find the genetic roots of rare hereditary pediatric diseases. These novel methods are not panaceas, however, and they often give ambiguous results, highlighting multiple possible causative mutations in affected patients. Furthermore, even when the mapping results are unambiguous, the affected gene might be of unknown function. In these cases, understanding how a particular genotype can result in a phenotype also needs carefully designed experimental work. Model organism genetics can offer a straightforward experimental setup for hypothesis testing. Containing orthologs for over 80% of the genes involved in human diseases, zebrafish (Danio rerio) has emerged as one of the top disease models over the past decade. A plethora of genetic tools makes it easy to create mutations in almost any gene of the zebrafish genome and these mutant strains can be used in high-throughput preclinical screens for active molecules. As this small vertebrate species offers several other advantages as well, its popularity in biomedical research is bound to increase, with “aquarium to bedside” drug development pipelines taking a more prevalent role in the near future. View Full-Text
Keywords: zebrafish; rare disease models; drug screening zebrafish; rare disease models; drug screening

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Varga, M.; Ralbovszki, D.; Balogh, E.; Hamar, R.; Keszthelyi, M.; Tory, K. Zebrafish Models of Rare Hereditary Pediatric Diseases. Diseases 2018, 6, 43.

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