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Diseases
2018 ; 6
(2
): ? Nephropedia Template TP
gab.com Text
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English Wikipedia
Zebrafish Models of Rare Hereditary Pediatric Diseases
#MMPMID29789451
Varga M
; Ralbovszki D
; Balogh E
; Hamar R
; Keszthelyi M
; Tory K
Diseases
2018[May]; 6
(2
): ? PMID29789451
show ga
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.
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