Model Organisms in the Fight against Muscular Dystrophy: Lessons from Drosophila and Zebrafish

Molecules

Emilie Plantié²  Marta Migocka-Patrzaᐮk¹  Małgorzata Daczewska¹  Krzysztof Jagla² 

[1]Department of Animal Developmental Biology, Institute of Experimental Biology, University of Wroclaw, 21 Sienkiewicza Street, 50-335 Wroclaw, Poland

[2] E-Mails:

[3]GReD (Genetics, Reproduction and Development laboratory), INSERM U1103, CNRS UMR6293, University of Clermont-Ferrand, 28 place Henri-Dunant, 63000 Clermont-Ferrand, France

[4] E-Mail:

关键词: muscular dystrophies;    animal models;    Drosophila;    zebrafish;    pathophysiology;    therapeutic screens;   

DOI  :  10.3390/molecules20046237

来源: mdpi

【 摘 要 】

Muscular dystrophies (MD) are a heterogeneous group of genetic disorders that cause muscle weakness, abnormal contractions and muscle wasting, often leading to premature death. More than 30 types of MD have been described so far; those most thoroughly studied are Duchenne muscular dystrophy (DMD), myotonic dystrophy type 1 (DM1) and congenital MDs. Structurally, physiologically and biochemically, MDs affect different types of muscles and cause individual symptoms such that genetic and molecular pathways underlying their pathogenesis thus remain poorly understood. To improve our knowledge of how MD-caused muscle defects arise and to find efficacious therapeutic treatments, different animal models have been generated and applied. Among these, simple non-mammalian Drosophila and zebrafish models have proved most useful. This review discusses how zebrafish and Drosophila MD have helped to identify genetic determinants of MDs and design innovative therapeutic strategies with a special focus on DMD, DM1 and congenital MDs.

【 授权许可】

CC BY   
© 2015 by the authors; licensee MDPI, Basel, Switzerland.