Recent advances using zebrafish animal models for muscle disease drug discovery

Expert Opin Drug Discov. 2014 Sep;9(9):1033-45. doi: 10.1517/17460441.2014.927435. Epub 2014 Jun 14.

Abstract

Introduction: Animal models have enabled great progress in the discovery and understanding of pharmacological approaches for treating muscle diseases like Duchenne muscular dystrophy.

Areas covered: With this article, the author provides the reader with a description of the zebrafish animal model, which has been employed to identify and study pharmacological approaches to muscle disease. In particular, the author focuses on how both large-scale chemical screens and targeted drug treatment studies have established zebrafish as an important model for muscle disease drug discovery.

Expert opinion: There are a number of opportunities arising for the use of zebrafish models for further developing pharmacological approaches to muscle diseases, including studying drug combination therapies and utilizing genome editing to engineer zebrafish muscle disease models. It is the author's particular belief that the availability of a wide range of zebrafish transgenic strains for labeling immune cell types, combined with live imaging and drug treatment of muscle disease models, should allow for new elegant studies demonstrating how pharmacological approaches might influence inflammation and the immune response in muscle disease.

Keywords: Duchenne muscular dystrophy; birefringence; drug screen; muscle function; muscle structure; myopathy; zebrafish.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Animals, Genetically Modified
  • Disease Models, Animal*
  • Drug Discovery / methods*
  • Humans
  • Inflammation / drug therapy
  • Inflammation / etiology
  • Inflammation / physiopathology
  • Muscular Diseases / drug therapy*
  • Muscular Diseases / immunology
  • Muscular Diseases / physiopathology
  • Muscular Dystrophy, Duchenne / drug therapy
  • Muscular Dystrophy, Duchenne / physiopathology
  • Zebrafish / genetics