Invertebrate models of spinal muscular atrophy: Insights into mechanisms and potential therapeutics

Invertebrate genetic models with their tractable neuromuscular systems are effective vehicles for the study of human nerve and muscle disorders. This is exemplified by insights made into spinal muscular atrophy (SMA) using the fruit fly Drosophila melanogaster and the nematode worm Caenorhabditis elegans. For speed and economy, these invertebrates offer convenient, whole-organism platforms for genetic screening as well as RNA interference (RNAi) and chemical library screens, permitting the rapid testing of hypotheses related to disease mechanisms and the exploration of new therapeutic routes and drug candidates. Here, we discuss recent developments encompassing synaptic physiology, RNA processing, and screening of compound and genome-scale RNAi libraries, showcasing the importance of invertebrate SMA models. Editor's suggested further reading in BioEssays: SMN and Gemins: 'We are family' ... or are we? Abstract Drosophila melanogaster and Caenorhabditis elegans can be used as convenient model organisms to study the cellular and molecular mechanisms underlying the function of the SMN gene. © 2011 WILEY Periodicals, Inc.