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.
MRC Functional Genomics Unit, Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, UK.
Stuart J
SJ
Grice
James N
JN
Sleigh
Ji-Long
JL
Liu
David B
DB
Sattelle
D000818:Animals; D017173:Caenorhabditis elegans / Q000235:genetics*; D004195:Disease Models, Animal; D004331:Drosophila melanogaster / Q000235:genetics*; D005786:Gene Expression Regulation; D015723:Gene Library; D057166:High-Throughput Screening Assays; D006801:Humans; D009046:Motor Neurons / Q000166:cytology / Q000378:metabolism; D009134:Muscular Atrophy, Spinal / Q000235:genetics* / Q000378:metabolism / Q000628:therapy; D034622:RNA Interference; D054852:Small Molecule Libraries / Q000378:metabolism
D054852:Small Molecule Libraries