RGD Reference Report - Inhibition of synapse assembly in mammalian muscle in vivo by RNA interference. - Rat Genome Database

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Inhibition of synapse assembly in mammalian muscle in vivo by RNA interference.

Authors: Kong, XC  Barzaghi, P  Ruegg, MA 
Citation: Kong XC, etal., EMBO Rep. 2004 Feb;5(2):183-8. Epub 2004 Jan 16.
RGD ID: 2317108
Pubmed: PMID:14749715   (View Abstract at PubMed)
PMCID: PMC1298976   (View Article at PubMed Central)
DOI: DOI:10.1038/sj.embor.7400065   (Journal Full-text)

The formation of the vertebrate neuromuscular junction (NMJ) requires the receptor tyrosine kinase MuSK and the adaptor molecule rapsyn. Here, we report that the phenotypes of mice deficient in these two molecules can be reproduced by RNA interference (RNAi) in rat muscle in vivo. Specifically, double-stranded RNA (dsRNA) targeting MuSK and rapsyn inhibited the formation of the NMJ in rat muscle fibres in vivo, while dsRNA targeting nonessential proteins did not have any effect. Moreover, plasmids that trigger RNAi to MuSK induced the disassembly of existing NMJs. These results thus demonstrate for the first time the functionality of dsRNA in silencing endogenous genes in adult mammalian muscle in vivo. Moreover, they show that MuSK is also required for the maintenance of the NMJ, offering a mechanistic explanation for the myasthenia gravis caused by auto-antibodies to MuSK.

Gene Ontology Annotations    Click to see Annotation Detail View

Biological Process
TermQualifierEvidenceWithReferenceNotesSourceOriginal Reference(s)
positive regulation of synaptic assembly at neuromuscular junction  IMP 2317108 RGD 

Objects Annotated

Genes (Rattus norvegicus)
Musk  (muscle associated receptor tyrosine kinase)


Additional Information