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 el...

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Main Authors: Grice, S, Sleigh, J, Liu, J, Sattelle, D
Format: Journal article
Language:English
Published: 2011
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author Grice, S
Sleigh, J
Liu, J
Sattelle, D
author_facet Grice, S
Sleigh, J
Liu, J
Sattelle, D
author_sort Grice, S
collection OXFORD
description 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.
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spelling oxford-uuid:abc93232-3acc-463e-8e47-83cb02ef94b62022-03-27T03:24:14ZInvertebrate models of spinal muscular atrophy: insights into mechanisms and potential therapeutics.Journal articlehttp://purl.org/coar/resource_type/c_dcae04bcuuid:abc93232-3acc-463e-8e47-83cb02ef94b6EnglishSymplectic Elements at Oxford2011Grice, SSleigh, JLiu, JSattelle, DInvertebrate 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.
spellingShingle Grice, S
Sleigh, J
Liu, J
Sattelle, D
Invertebrate models of spinal muscular atrophy: insights into mechanisms and potential therapeutics.
title Invertebrate models of spinal muscular atrophy: insights into mechanisms and potential therapeutics.
title_full Invertebrate models of spinal muscular atrophy: insights into mechanisms and potential therapeutics.
title_fullStr Invertebrate models of spinal muscular atrophy: insights into mechanisms and potential therapeutics.
title_full_unstemmed Invertebrate models of spinal muscular atrophy: insights into mechanisms and potential therapeutics.
title_short Invertebrate models of spinal muscular atrophy: insights into mechanisms and potential therapeutics.
title_sort invertebrate models of spinal muscular atrophy insights into mechanisms and potential therapeutics
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