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Invertebrate models of spinal muscular atrophy: Insights into mechanisms and potential therapeutics
Author(s) -
Grice Stuart J.,
Sleigh James N.,
Liu JiLong,
Sattelle David B.
Publication year - 2011
Publication title -
bioessays
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.175
H-Index - 184
eISSN - 1521-1878
pISSN - 0265-9247
DOI - 10.1002/bies.201100082
Subject(s) - spinal muscular atrophy , rna interference , caenorhabditis elegans , model organism , biology , drosophila melanogaster , invertebrate , genome , neuroscience , computational biology , organism , drosophila (subgenus) , drug discovery , disease , rna , genetics , bioinformatics , ecology , gene , medicine , pathology
Abstract 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