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Selective cleavage of AChR cRNAs harbouring mutations underlying the slow channel myasthenic syndrome by hammerhead ribozymes

Slow channel congenital myasthenic syndrome (SCCMS) is a dominant disorder caused by missense mutations in muscle acetylcholine receptors (AChR). Expression from mutant alleles causes prolonged AChR ion-channel activations. This ‘gain of function’ results in excitotoxic damage due to excess entry of...

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Detalhes bibliográficos
Main Authors: Abdelgany, Amr, Ealing, John, Wood, Matthew, Beeson, David
Formato: Artigo
Idioma:Inglês
Publicado em: Library Publishing Media 2005
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC2737193/
https://ncbi.nlm.nih.gov/pubmed/19771201
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