siRNA-mediated Allele-specific Silencing of a COL6A3 Mutation in a Cellular Model of Dominant Ullrich Muscular Dystrophy

Congenital muscular dystrophy type Ullrich (UCMD) is a severe disorder of early childhood onset for which currently there is no effective treatment. UCMD commonly is caused by dominant-negative mutations in the genes coding for collagen type VI, a major microfibrillar component of the extracellular...

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Detalles Bibliográficos
Autores principales: Bolduc, Véronique, Zou, Yaqun, Ko, Dayoung, Bönnemann, Carsten G
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2014
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3950771/
https://www.ncbi.nlm.nih.gov/pubmed/24518369
http://dx.doi.org/10.1038/mtna.2013.74

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3950771/
https://www.ncbi.nlm.nih.gov/pubmed/24518369
http://dx.doi.org/10.1038/mtna.2013.74

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