Swimming into prominence: the zebrafish as a valuable tool for studying human myopathies and muscular dystrophies

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A new and exciting phase of muscle disease research has recently been entered. The application of next generation sequencing technology has spurred an unprecedented era of gene discovery for both myopathies and muscular dystrophies. Gene-based therapies for Duchenne muscular dystrophy have entered clinical trial, and several pathway-based therapies are doing so as well for a handful of muscle diseases. While many factors have aided the extraordinary developments in gene discovery and therapy development, the zebrafish model system has emerged as a vital tool in these advancements. In this review, we will highlight how the zebrafish has greatly aided in the identification of new muscle disease genes and in the recognition of novel therapeutic strategies. We will start with a general introduction to the zebrafish as a model, discuss the ways in which muscle disease can be modeled and analyzed in the fish, and conclude with observations from recent studies that highlight the power of the fish as a research tool for muscle disease.

Zebrafish models of muscular dystrophies and myopathies recapitulate the major features of the corresponding human disorders, and have proven exceptionally useful for elucidating pathogenic mechanisms and identifying novel molecular therapies. In this review, we describe the features that make zebrafish an ideal system for studying muscle disease, and highlight key contributions they have made to our understanding of muscle disorders.

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