Editing Zebrafish (section)

=== Muscular dystrophies ===
[[Muscular dystrophy|Muscular dystrophies]] (MD) are a heterogeneous group of genetic disorders that cause muscle weakness, abnormal contractions and muscle wasting, often leading to premature death. Zebrafish is widely used as model organism to study muscular dystrophies.<ref name="Model organisms in the fight agains"/> For example, the ''sapje'' (''sap'') mutant is the zebrafish orthologue of human [[Duchenne muscular dystrophy]] (DMD).<ref>{{cite journal |vauthors=Kunkel LM, Bachrach E, Bennett RR, Guyon J, Steffen L |title=Diagnosis and cell-based therapy for Duchenne muscular dystrophy in humans, mice, and zebrafish |language=En |journal=[[Journal of Human Genetics]] |volume=51 |issue=5 |pages=397–406 |date=May 2006 |pmid=16583129 |pmc=3518425 |doi=10.1007/s10038-006-0374-9}}</ref> The Machuca-Tzili and co-workers applied zebrafish to determine the role of alternative splicing factor, MBNL, in [[Myotonic dystrophy|myotonic dystrophy type 1]] (DM1) pathogenesis.<ref>{{cite journal |vauthors=Machuca-Tzili LE, Buxton S, Thorpe A, Timson CM, Wigmore P, Luther PK, Brook JD |title=Zebrafish deficient for Muscleblind-like 2 exhibit features of myotonic dystrophy |journal=Disease Models & Mechanisms |volume=4 |issue=3 |pages=381–392 |date=May 2011 |pmid=21303839 |pmc=3097459 |doi=10.1242/dmm.004150}}</ref> More recently, Todd et al. described a new zebrafish model designed to explore the impact of CUG repeat expression during early development in DM1 disease.<ref>{{cite journal |vauthors=Todd PK, Ackall FY, Hur J, Sharma K, Paulson HL, Dowling JJ |title=Transcriptional changes and developmental abnormalities in a zebrafish model of myotonic dystrophy type 1 |journal=Disease Models & Mechanisms |volume=7 |issue=1 |pages=143–155 |date=January 2014 |pmid=24092878 |pmc=3882056 |doi=10.1242/dmm.012427}}</ref> Zebrafish is also an excellent animal model to study congenital muscular dystrophies including CMD Type 1 A (CMD 1A) caused by mutation in the human laminin α2 (LAMA2) gene.<ref>{{cite journal |vauthors=Jones KJ, Morgan G, Johnston H, Tobias V, Ouvrier RA, Wilkinson I, North KN |title=The expanding phenotype of laminin alpha2 chain (merosin) abnormalities: case series and review |journal=Journal of Medical Genetics |volume=38 |issue=10 |pages=649–657 |date=October 2001 |pmid=11584042 |pmc=1734735 |doi=10.1136/jmg.38.10.649}}</ref> The zebrafish, because of its advantages discussed above, and in particular the ability of zebrafish embryos to absorb chemicals, has become a model of choice in screening and testing new drugs against muscular dystrophies.<ref>{{cite journal |vauthors=Maves L |title=Recent advances using zebrafish animal models for muscle disease drug discovery |journal=Expert Opinion on Drug Discovery |volume=9 |issue=9 |pages=1033–1045 |date=September 2014 |pmid=24931439 |pmc=4697731 |doi=10.1517/17460441.2014.927435}}</ref>