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Allele-Specific Gene Silencing in Two Mouse Models of Autosomal Dominant Skeletal Myopathy

We explored the potential of mutant allele-specific gene silencing (ASGS) in providing therapeutic benefit in two established mouse models of the autosomal dominantly-inherited muscle disorders, Malignant Hyperthermia (MH) and Central Core Disease (CCD). Candidate ASGS siRNAs were designed and valid...

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Detalles Bibliográficos
Autores principales:	Loy, Ryan E., Lueck, John D., Mostajo-Radji, Mohammed A., Carrell, Ellie M., Dirksen, Robert T.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Public Library of Science 2012
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3495761/ https://www.ncbi.nlm.nih.gov/pubmed/23152933 http://dx.doi.org/10.1371/journal.pone.0049757

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